Focusing efforts on key health products to achieve the greatest public health impact in LMICs
The mission of the Medicines Patent Pool (MPP) is to facilitate the development of- and increase access to, life-saving medicines and health technologies for low- and middle-income countries (LMICs) through public-health oriented voluntary licensing and technology transfer. To do so, the first step for MPP is to identify suitable candidates using a prioritisation framework that is applied to assess products of potential interest in all health areas.
MPP’s list of priority and watchlist products is regularly updated, by following a peer-reviewed prioritisation framework. It outlines the health areas on which MPP focuses its activities.
MPP’s prioritisation list includes medicines and health technologies for which expanded access in LMICs could provide significant health benefits over standards of care, and where a voluntary agreement, including licensing and/or technology transfer, through MPP could lead to substantial public health impact.
This prioritisation process contributes to ensuring MPP focuses its efforts on medicines for which licensing could have the greatest public health impact. By using a robust framework, MPP evaluates product candidates and publishes a list of prioritised and watchlist medicines and health innovations in a range of health areas, for which a potential MPP intervention may support increased access.
It should be noted that MPP does not include in its prioritisation list the medicines and health innovations for which it has already signed agreements. The list of MPP previously signed agreements to enable access are available on the “agreements with innovators” section of MPP’s website.
VIRAL HEPATITIS B
VIRAL HEPATITIS C
MULTIPLE CANCER INDICATIONS
LUNG CANCER
BREAST CANCER
CHRONIC LYMPHOCYTIC LEUKAEMIA
PROSTATE CANCER
OTHER CANCER INDICATIONS
In 2024, 40.8 million people were living with Human Immunodeficiency Virus (HIV) worldwide, with 31.6 million people accessing antiretroviral therapy. HIV paediatric care is stalling, with only 55% of children living with the virus accessing treatment, compared to 78% of adults living with HIV. With 1.3 million people acquiring HIV annually, prevention remains key to tackle the transmission. Although pre-exposure prophylaxis (PrEP) has proven effective in preventing HIV infection, its uptake remains insufficient to meet global targets.
These persistent gaps highlight the need for affordable, effective HIV medicines, especially for people living with HIV (PLHIV) in low- and middle-income countries (LMICs) where HIV is most prevalent. Medicines must also be available in the right formulations. Fixed-dose combinations and long-acting formulations can help to improve adherence, while age-appropriate treatments for children, adapted to different weights and developmental stages, are essential to improve care.
Since 2010, we have worked with leading HIV drug manufacturers, governments, international organisations, civil society, and affected communities to improve access to World Health Organization (WHO) prioritised and recommended medicines for PLHIV in LMICs. As of December 2025, MPP licensees have supplied 53 billion doses of DTG-based regimens in 129 countries. The cumulative volumes supplied until December 2025 are equivalent to treating 142 million PLHIV with DTG-based regimens for a year. It is estimated that in 2025 there were 26 million PLHIV on DTG-based treatments.
Alongside HIV treatment access, MPP has worked to increase access to HIV prevention tools and supported the diversification of prevention options. In 2022, MPP signed a voluntary licensing agreement with ViiV Healthcare for cabotegravir long-acting (LA) for HIV PrEP, which was complemented in 2025 with the addition of the treatment indication, perfectly aligning with updated WHO guidelines for HIV. This marked an important step in accelerating affordable and equitable access to the first approved injectable long-acting PrEP and treatment options recommended by WHO in up to 142 countries.
These efforts reflect broader interest in long-acting therapeutics for both PrEP and HIV treatment, given their potential benefits in terms of efficacy (including through support of improved adherence), and user experience (including convenience and discretion). At the same time, 2025 saw an unprecedented disruption in HIV services as a consequence of international funding cuts. This underscores the need for more effective HIV programmes in LMICs, and for continued efforts to develop and roll out HIV prevention and treatment innovations, including long-acting and ARV-sparing regimens, to reach more people and manage increasingly scarce resources more effectively.
More on MPP’s contribution in the HIV field
MK-8527 is an investigational nucleoside reverse transcriptase translocation inhibitor (NRTTI) being developed for the prevention of HIV. MK-8527 shares mechanistic similarities with islatravir, offering the potential for high potency and a strong genetic barrier to resistance. MK-8527 is a promising clinical candidate for HIV PrEP, being evaluated for once-monthly oral dosing. Two phase III trials (EXPRESSIVE-10 and EXPRESSIVE-11) are ongoing, to evaluate the use on MK-8527 11mg tablet as once monthly oral PrEP in individuals 16 years and older.
Patent & licence data in LMICs
MK-8527 on LAPaL
Cabotegravir 4-monthly, also called CAB-ULA (ultra long-acting) is an investigational longer-acting formulation of cabotegravir that demonstrates a pharmacokinetic (PK) profile supporting dosing intervals of approximately four months. This extended dosing schedule has the potential to reduce clinic visits and improve adherence. Early data suggest a favourable safety profile, with intramuscular (IM) administration being better tolerated than subcutaneous (SQ) injection. As a result, CAB-ULA IM dosed every four months is advancing into late-stage studies investigating its use for both HIV-1 PrEP and treatment.
For PrEP, the EXTEND 4M registration trial is evaluating a single IM injection of CAB-ULA at 1600 mg/3mL. The FDA has approved this registration strategy, based on PK bridging studies, without requiring an additional efficacy study. The trial was launched in December 2024 and is expected to reach its primary endpoints by Q3 2026.
For HIV treatment, a phase III registration trial is evaluating CAB-ULA combined with rilpivirine (RPV) at Q4M dosing, following the completion of phase I studies on multiple formulations.
Primary patents on cabotegravir have been granted in many LMICs and are expected to expire in 2026. A secondary patent on the 4-monthly formulation was published in March 2025. The corresponding international patent application is pending, and geographical coverage in LMICs is expected to be available by March 2026, with patents anticipated to expire in 2043.
Cabotegravir 4 monthly on LAPaL
Patent information on cabotegravir 4-monthly
Islatravir/lenacapavir (ISL/LEN; MK-8591D) is an investigational once-weekly oral fixed-dose combination co-developed by MSD and Gilead for HIV treatment, pairing the first-in-class capsid inhibitor lenacapavir with the next-generation NRTTI islatravir. If approved, it would be the first long-acting oral HIV treatment taken once weekly, simplifying therapy and potentially improving adherence for virologically suppressed adults compared with daily oral regimens. On 8 June 2026, Gilead and MSD announced positive topline results from the pivotal Phase 3 ISLEND-1 and ISLEND-2 switch studies: both met their primary efficacy endpoint at Week 48.
ISL/LEN on LAPaL
Cabotegravir/lenacapavir (CAB/LEN) designates the investigational use of two separate long-acting formulations for HIV treatment, with or without rilpivirine, particularly for people with NNRTI resistance. It is of particular interest because it builds on drugs that already have generic versions in development and would not require a cold chain. Current generic licences for cabotegravir and lenacapavir do not cover this regimen.
Islatravir/ulonivirine (MK-8591B) is an investigational once-weekly oral fixed-dose combination of islatravir 2 mg and the NNRTI ulonivirine 200mg, developed by MSD as an alternative weekly oral treatment regimen to ISL/LEN.
VH-184 (aka VH4524184) is a third-generation INSTI, with a superior resistance profile. It could be important in cases of future INSTI resistance.
VH-184 on LAPaL
Islatravir/doravirine (DOR/ISL) is a once-daily oral fixed-dose combination of the NNRTI doravirine with the NRTTI islatravir (100 mg/0.25 mg), developed by MSD for virologically suppressed adults with HIV-1. It is the first non-INSTI-based two-drug regimen to demonstrate non-inferior efficacy and comparable safety to commonly used antiretroviral regimens in Phase 3 switch studies, with no treatment-emergent resistance and stable lymphocyte and CD4+ T-cell counts. On 21 April 2026 the U.S. FDA approved it as a switch option for adults who are virologically suppressed with no history of treatment failure and no known resistance to doravirine, making it the first FDA-approved two-drug regimen without an integrase inhibitor.
A yearly formulation of lenacapavir (3000 mg), given as two 3mL intramuscular injections with oral loading doses, is being investigated for HIV prevention, building on the approved twice-yearly subcutaneous formulation. Initial findings indicate it is safe and well tolerated and maintains plasma concentrations above the protective threshold for at least 56 weeks, with the Phase 3 PURPOSE 365 trial ongoing. By further extending the dosing interval, yearly lenacapavir could become a powerful additional PrEP tool if affordable and accessible.
VH-499 (VH4011499) is an investigational HIV-1 capsid inhibitor being developed by ViiV Healthcare as a long-acting injectable for HIV treatment. It belongs to the same mechanistic class as lenacapavir but is structurally distinct, notably lacking CYP3A4 inhibition or induction, a potential advantage for people with comorbidities or polypharmacy, such as those on rifampicin-based TB treatment. Early data show potent oral antiviral activity and an injectable profile supporting twice-yearly dosing.
GS-3242 is a novel investigational INSTI being developed by Gilead as a long-acting injectable for HIV-1 treatment, engineered for dosing intervals of at least four months. It retains activity against INSTI-resistant variants, with no apparent cross-resistance to other classes, and early clinical data support at least four-monthly dosing.
GS-1720/GS-4182 is an investigational once-weekly oral regimen for HIV treatment, combining the INSTI GS-1720 (lepetegravir) with GS-4182, an oral prodrug of lenacapavir. The Phase 2/3 WONDERS trials were placed on clinical hold in June 2025 after some participants developed low CD4+ T-cell counts and reduced total white blood cell counts. WONDERS 2 was terminated in April 2026, while WONDERS 1 remains on clinical hold under ongoing safety follow-up.
This insert is a candidate tenofovir alafenamide/elvitegravir (TAF/EVG) insert fast-dissolving, bullet-shaped tablet for vaginal or rectal administration for on-demand HIV prophylaxis (both PrEP and post-exposure prophylaxis, PEP). The insert contains a co-formulation of antiretroviral drugs that dissolve upon insertion, preventing HIV replication locally. This candidate passed several phase I studies. It is awaiting to enter phase II to continue assessing safety and effectiveness.
CONRAD have filed for patents on their TAF/EVG sustained release formulation for vaginal or rectal use in 7 LMICs, including South Africa. If granted, these would likely expire in 2039. If successful, this product could complement other PrEP and PEP options, serving clients of any gender for whom a discreet, user-initiated, event-driven HIV prevention method would be appealing. The manufacturing prospects indicate a potential low-price for quality assured generic versions if a voluntary licence agreement is reached.
These laboratory-made proteins emulate the immune system’s ability to counteract harmful pathogens, including HIV. Although still in the early stages of development, broadly-neutralising antibodies demonstrate significant potential for use as HIV post-natal prophylaxis (PNP), due to their favourable safety profile and the convenience they may offer through a single administration covering the peri-natal and breastfeeding period via intramuscular injection,,. Despite potential benefits, crucial clinical data on the efficacy of monoclonal antibodies for PNP remain lacking. Specifically, there is a notable gap in understanding whether a single monoclonal antibody could suffice for prevention. If multiple mAbs are necessary, the cost could become a significant concern, especially when compared to cheaper small molecules.
Beyond their use in postnatal prophylaxis, broadly neutralizing antibodies (bNAbs) are also being investigated for HIV treatment and cure strategies.
TB is present in all countries and age groups. An estimated 10.7 million people fell ill with tuberculosis (TB) in 2024, most of them in LMICs. TB remains among the top 10 causes of death worldwide and the leading cause of death from a single infectious agent. In 2024, an estimated 1.23 million people died from TB globally. TB is also the leading cause of death among PLHIV and a major contributor to antimicrobial resistance.
However, TB is curable and preventable.
Globally, there were an estimated 390 000 people who developed rifampicin-resistant TB (RR-TB) in 2024, of whom 164 545 received treatment, representing about 42% of those in need of treatment.
The End TB Strategy sets ambitious targets to reduce TB deaths by 95% between 2015 and 2035 and end the global TB epidemic. However, despite important advances, progress remains insufficient: between 2015 and 2024 TB deaths declined by 29% and the TB incidence rate fell by 12%, far short of the interim milestones.
To meet these targets, better TB products for both treatment and prevention are still urgently needed, particularly for drug-resistant TB. Current regimens still fall short on several fronts. They need to be shorter, safer, better tolerated, more resilient to resistance, and compatible with HIV co-treatment. They also need to be better adapted to children and other populations that remain poorly served by current TB programmes. In the future, long-acting approaches could also help simplify treatment, improve adherence, and expand prevention options.
At MPP, we work to improve access to new treatments for MDR-TB and drug-susceptible tuberculosis, as well as facilitating development of innovative combinations allowing for shorter/optimized treatment courses, including for tuberculosis preventative therapies (TPT), by licensing TB drugs that are still under development.
In early 2017, MPP signed its first agreement with the Johns Hopkins University 2. This agreement was to facilitate the clinical development of sutezolid, a promising investigational treatment for tuberculosis. It was followed by a second agreement with Pfizer in October 2019 to access Pfizer’s preclinical, phase I and phase IIa clinical study data and results on sutezolid 3. The agreement’s aim was to further study, develop and make available this potential important component of new TB regimens.
Moreover, in 2021, MPP signed the first non-exclusive, worldwide, voluntary license agreement with Tandem Nano, a spin-off of University of Liverpool, for a long-acting TPT candidate, using injectable formulations of rifapentine and an isoniazid prodrug, developed by members of the LONGEVITY consortium, funded by Unitaid.
Quabodepistat (previously known as OPC-167832) is a medicine with a new mechanism of action that has potent antituberculosis activity and a favourable safety profile. It is an inhibitor of DprE1, an enzyme essential to the Mycobacterium tuberculosis cell wall, giving it a novel mechanism of action with high potency against both drug-susceptible and drug-resistant TB. In a Phase 2b/c trial, a four-month quabodepistat regimen combined with delamanid and bedaquiline achieved safety and efficacy broadly similar to the standard six-month four-drug regimen in drug-susceptible TB, although exploratory follow-up suggested that a more potent fourth drug may be needed to reliably shorten treatment. It is now being evaluated within regimen-based strategies, including the PARADIGM4TB platform trial and the Phase 3 QUANTUM trial, which tests a quabodepistat-containing regimen designed to replace the more toxic linezolid, with primary completion expected in May 2027. As a treatment-shortening candidate well suited to the MPP access model, it remains a priority.
Alpibectir (AlpE) is an investigational oral agent with a novel mechanism: it activates bacterial pathways that boost the activity of ethionamide, an older anti-TB drug whose use is normally limited by dose-related toxicity. By enabling ethionamide to work at much lower, better-tolerated doses, alpibectir could improve both efficacy and safety. In a Phase 2 early bactericidal activity study in South Africa, low-dose ethionamide combined with alpibectir matched the activity of high-dose ethionamide and isoniazid while reducing side effects, and further dose-finding (ENABLE) and UNITE4TB regimen studies are ongoing, with later readouts expected through 2027. It holds orphan-drug designation from both the FDA and EMA. Primary patents on alpibectir have been filed in many LMICs and are expected to expire in 2038.
Bedaquiline is a crucial antibiotic for treating TB, particularly MDR-TB. It significantly improves treatment outcomes by reducing mortality and shortening therapy duration compared to older, less effective, often injectable, regimens. A long-acting intramuscular formulation is being developed by J&J as a single injection for TB preventive treatment (TPT), where poor adherence to current multi-week oral regimens is a major barrier. TPT is recommended by WHO for people living with HIV, household contacts of people with TB, and other risk groups. A Phase 1 study, with recruitment complete and results expected by the end of 2026, aims to identify a dose with pharmacodynamics at least comparable to the 3HP regimen (three months of weekly isoniazid plus rifapentine). Although the compound patent expired in 2023, Janssen owns several patents in LMICs on long-acting formulations of bedaquiline expiring between 2038 and 2041.
Sudapyridine (WX-081) is a new oral ATP synthase inhibitor with a mechanism similar to bedaquiline, of particular interest for MDR-TB. It has the potential to reduce some of the drawbacks associated with bedaquiline, such as QT prolongation, liver toxicity and gastrointestinal effects. A registrational Phase 3 trial in China is comparing sudapyridine against bedaquiline, each added to a background regimen, in rifampicin-resistant pulmonary TB, with results expected in October 2026. It remains on the watchlist while clinical data are still limited. Primary patents on sudapyridine are present in key countries of manufacture such as India, China and South Africa and are expected to expire in 2035. Secondary patents covering manufacturing processes and intermediates with an expected expiry date in 2037 are present in a few LMICs.
Macozinone (PBTZ169) is an investigational oral DprE1 inhibitor developed by the non-profit iM4TB, with high preclinical potency against both drug-susceptible and drug-resistant TB. Phase 1 studies confirmed a favourable safety and tolerability profile. Macozinone has additive effects with many TB therapeutic agents, both marketed and in development, and has synergic effects with bedaquiline and clofazimine in preclinical models. There is a long-acting injectable formulation in early development with MedinCell through the EU ERA4TB programme. Primary patents on macozinone have been granted in key countries of manufacture such as India, China and South Africa and are expected to expire in 2031. Secondary patents may be filed.
Ganfeborole (GSK3036656) is an investigational oral agent and a potential first-in-class TB drug, targeting leucyl-tRNA synthetase, a wholly new mechanism that could support the shorter, more effective regimens needed particularly for drug-resistant TB. Phase 2a studies have shown measurable early bactericidal activity with good tolerability, and it is being evaluated in two-drug combinations and within the UNITE4TB M4TB platform trial, expected to complete in August 2027. A key limitation is preclinical evidence of teratogenicity, which may make it unsuitable for pregnant women and could leave a gap in the goal of a single universal "Pan-TB" regimen. Patents on the compound have been filed or granted in several LMICs and are expected to expire between 2031-2036. Additional secondary patents may be filed.
Delpazolid is an investigational oral oxazolidinone being developed for TB, in the same class as linezolid but with the aim of avoiding linezolid's long-term toxicities. It is being studied in combination with delamanid and bedaquiline. n the Phase 2b PanACEA-DECODE-01 dose-finding trial, added to a backbone of bedaquiline, delamanid and moxifloxacin, pharmacokinetic-pharmacodynamic modelling suggested that it adds efficacy and that 1200 mg once daily would maximise efficacy while remaining safe, raising the prospect of averting linezolid-type toxicity in long-term treatment. The primary patent on delpazolid is filed or granted in key countries of manufacture such as India, China and South Africa and is expected to expire in 2029. Secondary patents with an expected expiry date in 2031 are filed or granted in a few LMICs.
In 2022, viral hepatitis claimed approximately 1.3 million lives, of which 83% were attributed to hepatitis B and 17% to hepatitis C. New infections decreased from 3 million in 2019 to 2.2 million in 2022 (1.2 million hepatitis B, 1.0 million hepatitis C). Despite this progress, the global burden remains substantial with 304 million people living with viral hepatitis in 2022 (254 million with hepatitis B, 50 million with hepatitis C).
Children represent 12% of the hepatitis burden, primarily affecting those with hepatitis B. Significant regional disparities persist, with the WHO African Region accounting for 63% of new hepatitis B infections, yet only 18% of newborns in this region receive the hepatitis B birth-dose vaccine. The Western Pacific Region reports 47% of hepatitis B deaths, with inadequate treatment coverage.
Each form of viral hepatitis (B, C and D) presents distinct clinical unmet needs and disease-specific considerations that are important to take into account when assessing product relevance and access pathways for LMICs. At the same time, the three diseases are linked by common access challenges, particularly underdiagnosis, limited screening, and the difficulty of delivering affordable care at scale.
Although hepatitis C is curable, there is an urgent need for well-tolerated, highly effective pangenotypic cure regimens requiring less frequent administration. The identification of populations living with the virus remains challenging due to limited awareness and screening programs. High-cost diagnostic tools and the requirement for confirmatory molecular diagnostics represent key barriers to treatment access.
Hepatitis B, while preventable through vaccination, maintains high prevalence rates. Beyond improved diagnostics, treatments that could provide a functional cure for hepatitis B are essential to reduce long-term disease burden, management costs, and complications. Expanded prevention, screening, and treatment initiatives are critical to address the global hepatitis crisis. Currently, few LMICs have committed to hepatitis elimination goals, and additional funding for effective diagnostic and care implementation is urgently needed.
This is even more critical for hepatitis D, which remains an area of high unmet medical need, with very limited treatment options and a substantial risk of severe liver outcomes, including cirrhosis and liver cancer. Occurring only in people living with hepatitis B, hepatitis D is associated with a two- to six-fold higher risk of liver cancer compared with hepatitis B alone. However, despite encouraging progress, future treatment approaches will still face several important challenges, notably ease of administration, long-term efficacy, and persistent diagnostic barriers, which may further complicate implementation, especially in LMICs.
MPP collaborates with diverse stakeholders, including originator and generic companies, governments, WHO, civil society, communities, procurement agencies, and clinical experts associations, to accelerate development and distribution of new treatments for eliminating HCV through shorter, more effective therapies in LMICs with high viral hepatitis burden. MPP’s multilateral engagements increase awareness of WHO-recommended treatment options available through MPP licenses (including TDF, TAF, DAC, and DAC/SOF combinations) and support increased uptake of quality-assured, affordable treatments in LMICs.
Notably, MPP’s work also addresses HBV through licensed TDF and TAF treatments, benefiting people living with HIV who receive TDF and TAF-based regimens with diagnosed or undiagnosed HBV, as well as individuals using oral PrEP containing TDF or TAF. Through these licenses, MPP supports the WHO strategy for triple elimination of vertical transmission of HIV, syphilis, and hepatitis B.
MPP closely monitors the evolving viral hepatitis therapeutics R&D pipeline (for hepatitis B, C, and D) , in collaboration with leading experts to assess potential MPP interventions for leading candidates. Currently, three medicines (including investigational medicines) are included in the watchlist. With the currently available data, no viral hepatitis therapies have been identified as priority candidates for which an MPP agreement would achieve significant impact.
Bepirovirsen is an investigational antisense oligonucleotide (ASO) therapy being developed for chronic hepatitis B. It targets all HBV messenger RNAs through a triple-action mechanism: (1) by reducing HBV replication, (2) by suppressing hepatitis B surface antigen (HBsAg) levels, and (3) by stimulating the immune system to potentially achieve a functional cure.
It is studied as weekly subcutaneous injections over a finite 24-week course. In the pivotal Phase 3 B-Well 1 and B-Well 2 trials, presented at EASL 2026, around one in five virologically suppressed, non-cirrhotic adults on stable nucleos(t)ide analogue therapy achieved a functional cure, defined as sustained HBsAg loss with suppressed HBV DNA after stopping all treatment, compared with none on placebo, with the strongest response in those with lower baseline HBsAg (functional cure of roughly 25 to 28% at HBsAg ≤1000 IU/mL). This makes it the most advanced finite-duration functional-cure candidate for hepatitis B, a meaningful advance over current therapies that generally require lifelong dosing to maintain suppression. It is not a universal cure: the evidence supports a defined subgroup of NA-suppressed, non-cirrhotic patients with HBsAg ≤3000 IU/mL, and implementation in LMICs will depend on access to quantitative HBsAg testing, structured monitoring for ALT, platelet and renal changes, affordability and ASO manufacturing feasibility.
GSK in-licensed bepirovirsen from Ionis (formerly Isis) Pharmaceuticals in 2019. Ionis holds the primary patent on bepirovirsen, which has been filed in many low- and middle-income countries (LMICs) and is expected to expire in 2032.
Bemnifosbuvir/ruzasvir is an investigational once-daily oral, pan-genotypic fixed-dose combination for chronic hepatitis C, pairing a nucleotide polymerase inhibitor (bemnifosbuvir) with an NS5A inhibitor (ruzasvir). In Phase 2, an 8-week course achieved high cure rates, with SVR12 of 98% in treatment-adherent patients and 99% in non-cirrhotic patients, although lower at 88% in those with compensated cirrhosis, alongside a favourable safety profile, no food effect and a low risk of drug-drug interactions. Two open-label Phase 3 trials, C-BEYOND (North America) and C-FORWARD (outside North America), are comparing it against sofosbuvir/velpatasvir, with topline results expected in mid-2026 and December 2026 respectively. Its potential advantages are a shorter 8-week treatment duration and efficacy across HCV genotypes 1 to 4, but because effective and relatively affordable pan-genotypic cure regimens already exist, its incremental benefit over the current standard of care is uncertain, so it remains on the watchlist. Atea’s primary patents on bemnifosbuvir, expected to expire in 2036, have been granted in 35 LMICs and are pending in another 14 LMICs. Atea owns several secondary patents on salts and polymorphs that may provide exclusivity until 2038-2042. Ruzasvir was first developed by MSD and exclusively licensed to Atea in 2021. Primary patents, expected to expire between 2031 and 2033, have been widely withdrawn and kept in force only in a few European countries. Atea filed two combination patents for bemnifosbuvir + ruzasvir. The second combination patent, expiring in 2042, is pending in 17 LMICs.
Malaria remains a major global public health challenge, with an estimated 282 million cases and approximately 610 000 deaths globally in 2024, the vast majority occurring in sub-Saharan Africa. This represents a slight increase compared to 2023, highlighting a stagnation of progress in malaria control. Children under five years of age continue to bear a disproportionate burden, accounting for approximately 75–80% of malaria-related deaths in the African region. Despite significant progress over the past two decades, recent trends indicate a stalling of progress, highlighting the need for renewed efforts and innovative interventions.
WHO guidelines emphasize a comprehensive, integrated approach to malaria control, combining vector control (e.g. insecticide-treated nets and indoor residual spraying), preventive chemotherapies, vaccination, and effective case management. In parallel, WHO now recommends the use of malaria vaccines in children living in endemic areas as part of a broader package of interventions, marking a significant shift in malaria prevention strategies.
At the same time, drug resistance remains a key threat, reinforcing the need to develop new treatment options and optimize existing therapies. In this context, the pipeline for antimalarial medicines has strengthened in recent years, including new mechanisms of action and simplified regimens aimed at improving adherence and treatment outcomes.
A critical gap identified by WHO is the lack of age-appropriate paediatric formulations. The PADO malaria exercise (2025) highlights that the development of medicines for children continues to lag significantly behind that for adults, contributing to suboptimal treatment outcomes and adherence challenges. Many existing antimalarial medicines remain poorly adapted for children due to issues such as bitter taste, complex dosing, and limited flexibility, which can lead to incomplete treatment and increased risk of resistance.
The MicroCoat platform technology employs a fluid bed coating process to produce individually coated drug micropellets, that regulate the rate of drug release, enabling taste masking and extended release as relevant. The technology is investigated for an application to a malaria treatment for paediatric populations in LMICs through the support of a UnitaidExplore grant . MicroCoat AS/AQ combines into a fixed-dose combination of taste-masked anti-malarial drugs artesunate and amodiaquine, offering ease of administration and improved palatability for children compared to marketed products (especially for amodiaquine, which is very bitter and thereby challenging for use in children), while maintaining the same efficacy and safety. The formulation has been designed to be suitable for infants and young children for the treatment of uncomplicated P. falciparum malaria in LMICs, by ensuring not only palatability, but also easier swallowing and more flexible dosing than standard of care. The product is stable at accelerated and long-term conditions and is in early clinical development.
As the product has been included in the first Paediatric Drug Optimization (PADO) priority list for malaria therapies for children in 2025, highlighting its importance in children and in LMICs, the product is now a priority.
The University of Hertfordshire, from which Fluid Pharma spun out, has filed patents relating to the coating process, with an expected expiry in 2038. Patents have been granted in the United States and China, and a corresponding application is pending in Europe.
MPP has prioritised Fluid Pharma’s product because it addresses a key gap in paediatric malaria formulations and presents an opportunity to accelerate its development and access through technology transfer.
Respiratory syncytial virus (RSV) is a leading cause of acute lower respiratory infections in children and severe respiratory disease in the elderly. Each year, RSV leads to over 3.6 million hospitalisations and around 100,000 deaths in children under 5, with 97% of paediatric deaths occurring in low- and middle-income countries due to limited medical care. While most young children have mild symptoms, some, especially those with their first infection or underlying health conditions, develop severe diseases like pneumonia and bronchiolitis. Two immunisation options are available for infants: a monoclonal antibody and a maternal vaccine. Three vaccines also prevent severe RSV in elderly adults with certain health conditions. However, the high cost of these vaccines and under-recognition of RSV’s impact in LMICs delay access to these potentially life-saving interventions. Therefore, there is a delay in introducing these potentially life-saving interventions for RSV prevention into the countries where they are most needed.
Nirsevimab is a monoclonal antibody with activity against Respiratory Syncytial Virus (RSV). Monoclonal antibodies are laboratory-made proteins that mimic the immune system’s ability to fight off harmful pathogens such as viruses. One dose of nirsevimab administered to infants as a single intramuscular injection prior to, or during RSV season, may provide protection during the RSV season. Infants have the highest incidence of severe disease, peaking at 1–3 months of age.
Nirsevimab on MedsPaL
Nirsevimab on LAPaL
Clesrovimab is an investigational mAb, which in phase III trials was shown to be very effective in preventing RSV-associated hospitalisations and LRTI when administered as a single dose to infants of all weights from birth to 1 year, and entering their first season of RSV.
Patents covering clesrovimab have been filed or granted in more than fifty LMICs and they are expected to expire in 2036. Secondary patents covering a formulation expected to expire 2039 were filed in few LMICs. Other secondary patents may be filed.
Acronyms & abbreviations list
Clesrovimab on MedsPaL
Clesrovimab on LAPaL
Retavibart (formerly TNM001) is a long-acting monoclonal antibody targeting the RSV prefusion F protein, designed to provide seasonal protection after a single intramuscular dose in infants, including both healthy and high-risk children entering their first RSV season. In a Phase 2b placebo-controlled trial, a single 120 mg dose was well tolerated and reduced medically attended RSV lower respiratory infection by around 66% and RSV-associated hospitalisation by around 82% versus placebo, with greater efficacy suggested in high-risk infants from a limited dataset. A Phase 3 trial has closed and Trinomab is filing with China's NMPA. It is included on the PADO RSV watch list.
Ziresovir is a novel oral, small-molecule RSV fusion (F) protein inhibitor being developed to treat RSV infection in infants and young children, dosed by body weight. It is in the preregistration phase in China, and would address an unmet need given that aerosolised ribavirin is currently the only approved RSV antiviral. In the Phase 3 AIRFLO trial in hospitalised infants and children, where it was given twice daily for five days, ziresovir achieved a significantly greater reduction in Wang bronchiolitis clinical score at day 3 and in RSV viral load at day 5 than placebo, with no major safety concerns, although resistance-associated mutations emerged in around 9% of treated participants. The WHO PADO group regards it as the most advanced oral RSV antiviral, but flags limitations including an endpoint not validated specifically for RSV, the uncertain clinical meaningfulness of viral-load endpoints, relatively late recruitment, and a China-only study population.
Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, or nearly one in six deaths1. Among the prevalent cancer types, breast, lung, colon and rectum, prostate, stomach, liver, cervix uteri and skin cancers stood out as the most frequently encountered. Many cancers can be cured if detected early and treated effectively. Many health systems in low- and middle-income countries remain ill-equipped to cope with this growing health crisis. Consequently, a significant portion of cancer patients worldwide continues to face significant barriers to accessing timely and high-quality diagnosis and treatment.
Lung cancer arises from the rapid and unregulated proliferation of abnormal cells in the lungs, posing a significant threat to health and carrying a high risk of fatality. The two most common forms of lung cancer are: non-small cell lung carcinoma (NSCLC), which is prevalent and tends to develop at a gradual pace, and small cell lung carcinoma (SCLC), which is rarer but usually exhibits a rapid growth rate. In low- and middle-income countries (LMICs), there were 1.2 million incident cases of non-small cell lung cancer (NSCLC) in 20202. Unfortunately, around 70% of NSCLC cases are diagnosed in advanced stages, such as locally advanced or metastatic.
Breast cancer is the most commonly occurring cancer in women and the most common cancer overall. There were more than 2.26 million new cases of breast cancer in women in 20203. There are four main female breast cancer subtypes, including the following in order of prevalence: HR+/HER2-, HR-/HER2-, HR+/HER2+, HR-/HER2+. HR stands for hormone receptor. HR+ means that tumour cells have receptors for the hormones estrogen or progesterone, which can promote the growth of HR+ tumours. HER2 stands for human epidermal growth factor receptor 2. HER2+ means that tumour cells make high levels of a protein called HER2/neu, which has been shown to be associated with certain aggressive types of breast cancer4.
Chronic Lymphocytic Leukaemia (CLL) is the most common form of leukaemia, accounting for 25% to 30% of all leukaemia cases in Western countries5. In 2019, CLL resulted in 44,612 deaths worldwide. Regarding incidence rates in 2019, CLL affected 1.34 individuals per 100,000 in the global population, with rates of 1.13 in high-income countries, 0.45 in middle-income countries, and 0.28 in low-income countries6. CLL predominantly occurs in older individuals, peaking in the elderly population, with a median age at diagnosis of 71 years in Europe7. Furthermore, the incidence of CLL is approximately twice as high in males compared to females8.
Prostate cancer made up 7.3% of all new cancer cases in 2020, accounting for 14.1% of all new male cancer diagnoses9. Notably, cancer incidence rates have been on the rise in various Sub-Saharan African populations10. Men of African descent face nearly double the risk of being diagnosed with prostate cancer before the age of 45 compared to Caucasian men11. Additionally, research indicates that men with greater overall and central body fat have a heightened risk of prostate cancer-related mortality12.
Immune checkpoint inhibitors (ICIs) have revolutionised oncology, opening new possibilities for cancer treatment through immunotherapy. These monoclonal antibodies block proteins that would otherwise prevent the immune system from attacking cancer cells. The most well-known in this class are PD-1 and PD-L1 inhibitors. Their versatility has led to approval for a wide range of cancers, with a robust and promising pipeline of new ICIs. The WHO EML Committee has recognised their therapeutic value by including certain ICIs for the treatment of cutaneous melanoma, underscoring their effectiveness and potential for broader application.
Notably, ICIs have shown promise in addressing some of the most pressing global cancer challenges, including breast and cervical cancers—the most commonly diagnosed and deadliest cancers among women in LMICs. Among people living with HIV, many cancers occur more frequently due to infectious origins, such as Kaposi’s sarcoma (linked to human herpesvirus 8), non-Hodgkin lymphoma (linked to Epstein–Barr virus), and cervical cancer (linked to human papillomavirus, HPV) . People living with HIV who are on antiretroviral therapy and virally suppressed can live longer, managing HIV as a chronic condition, and face similar health issues as the general population. As a result, cancer has emerged as a leading cause of death in this population, posing new clinical challenges. Despite the paucity of safety and efficacy data of ICIs in the HIV population, emerging studies suggest that ICIs treatments do not interfere with HIV management, reinforcing the value of these therapies for this population.
However, ensuring broad access to ICIs remains a challenge. Recognising this, the WHO EML Committee has recommended further efforts to improve the affordability of ICIs, including consideration of voluntary licensing through MPP. In addition to voluntary licensing of intellectual property, MPP’s support for hands-on technology transfer could help accelerate development and lower the costs of quality-assured biosimilar versions of ICIs for use in LMICs. In line with this, MPP has strategically prioritised ICIs as a class, with pembrolizumab, approved for 18 types of cancer, including certain early-stage and advanced cancers, identified as a flagship product for this class.
acronyms & abbreviations list
Pembrolizumab is an immune checkpoint inhibitor, specifically an anti-programmed death receptor-1 (PD-1) therapy, that enhances the body’s immune response to detect and combat tumour cells. It is a humanised monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes, which can target both tumour and healthy cells. Pembrolizumab as a single agent reduces the risk of death in NSCLC patients by 40%, significantly extending survival by more than a year with fewer sides effects compared to traditional chemotherapy . First approved in 2014, pembrolizumab has since gained approval for 18 types of cancer, including lung cancer and certain early-stage and advanced cancers. MSD holds the exclusive patent rights on pembrolizumab until 2028 with patents filed or granted in at least 15 LMICs.
Pembrolizumab on LAPaL
Aumolertinib is a third-generation molecular targeted drug that acts as an epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI) with antineoplastic activity. It inhibits mutant forms of EGFR, including the T790M mutation. Aumolertinib has demonstrated significant benefits over standard first- and second-generation treatments included in the WHO Essential Medicines List for NSCLC. It is currently under review for approval by the European Medicines Agency (EMA) . Aumolertinib compound patent is expected to expire in 2035, and it has been granted in key countries of manufacture such as India, China and South Africa. Secondary patents may provide exclusivity in few LMICs until 2036-2039.
Osimertinib is a third-generation molecular targeted drug that acts as an EGFR TKI, designed to target both EGFR-sensitising and EGFR T790M resistance mutations. It has demonstrated clinical activity against central nervous system (CNS) metastases and is approved by both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Osimertinib has shown significant benefits over standard first- and second-generation treatments included in the WHO EML for non-small cell lung cancer. The EML committee has called on MPP to explore licensing opportunities for osimertinib . The primary patent on osimertinib is expected to expire in 2032 and has been granted in more than 60 LMICs.
Adagrasib is an oral Kirsten Rat Sarcoma Virus (KRAS) inhibitor designed to potently and selectively target the KRAS G12C mutation. It received accelerated approval from the US FDA in 2022 for the treatment of NSCLC and has demonstrated durable clinical benefits, including improved progression-free survival . Notably, in 2024, the US FDA also granted accelerated approval for adagrasib in combination with immunotherapy for adults with KRAS G12C-mutated colorectal cancer, further reinforcing its potential across multiple tumour types. Adagrasib primary patents have been filed in many LMICs and are expected to expire in 2038.
Lazertinib is an oral, potent, and irreversible EGFR TKI that is highly selective for activating (EGFRm) and T790M resistance mutations. In 2024, the US FDA approved the combination of lazertinib and immunotherapy for first-line treatment of NSCLC harbouring the mutation. Research on lazertinib monotherapy is ongoing, highlighting its potential as an alternative treatment option for lung cancer. The primary patent on lazertinib has been granted in many LMICs including India and is expected to expire in 2034. There are secondary patents that may provide further exclusivity until 2038-2041 in many LMICs.
Sotorasib is an oral Kirsten Rat Sarcoma Virus (KRAS inhibitor designed to potently and selectively target the KRAS G12C mutation. It received accelerated approval from the US FDA in 2021 for the treatment of NSCLC. Notably, in January 2025, the FDA granted accelerated approval for sotorasib in combination with immunotherapy for adults with KRAS G12C-mutated colorectal cancer, further reinforcing its potential across multiple tumour types. The primary patent on sotorasib has been granted in many LMICs and is expected to expire in 2038. Several secondary patents on crystalline forms and processes are expected to expire between 2039 and 2040.
Abemaciclib is an oral cyclin-dependent kinase 4 and 6 (CDK 4/6) inhibitor, approved by the US FDA for treating HR+/HER2- advanced breast cancer . CDK 4/6 inhibitors are the preferred treatment option for HR+/HER2- advanced breast cancer. The EML expert committee recognised its potential for future inclusion and recommended that MPP explore licensing opportunities to support affordable access. Abemaciclib primary patents have been granted in more than 40 LMICs and are expected to expire in 2029 in most countries.
Ribociclib is an oral CDK 4/6 inhibitor, approved by the US FDA for treating HR+/HER2- advanced breast cancer . CDK 4/6 inhibitors are the preferred treatment option for HR+/HER2- advanced breast cancer. The EML expert committee recognised its potential for future inclusion and recommended that MPP explore licensing opportunities to support affordable access. Ribociclib and abemaciclib are similar alternative treatment options. Ribociclib primary patents have been filed or granted in many LMICs and are expected to expire between 2027 and 2029. Secondary patents may provide exclusivity until 2031-2036 in many LMICs.
Trastuzumab, a humanised IgG1 monoclonal antibody used to treat HER2-overexpressing metastatic breast cancer, has been developed (from infusion initially) into a subcutaneous formulation using recombinant human hyaluronidase to enhance delivery by temporarily breaking down hyaluronan, a barrier-forming substance in the tissues beneath the skin. Approved in 2019, the subcutaneous formulation follows a fixed-dose regimen, making it easier and quicker to administer than the original intravenous version. Trastuzumab primary patents expired in 2012. Secondary patents on the subcutaneous formulation have been filed and granted widely in LMICs and are expected to expire in 2030.
Zanubrutinib is a BTKi approved by the FDA in 2023 for the treatment of CLL. Recognizing the important emerging role of BTKi as a therapeutic class in the treatment of CLL, the EML Committee advised that it would consider an application for zanubrutinib as therapeutic alternative for inclusion and recommended that MPP explore licensing opportunities to support affordable access. The primary patent on zanubrutinib was granted in few LMICs, including India, where it is expected to expire in 2034. Secondary patents on crystalline forms are expected to expire in 2037.
Apalutamide is an oral second-generation androgen receptor antagonist approved by the US FDA in 2018. For the treatment of non-metastatic castration-resistant and metastatic castration-sensitive prostate cancer, clinical guidelines recommend androgen receptor antagonists, such as apalutamide, in combination with androgen-deprivation therapy as the standard of care. With approval for both of these advanced prostate cancer types, apalutamide emerges as a key therapeutic option. Apalutamide primary patents are expected to expire in 2027. Secondary patents may provide exclusivity until 2033-2038 in many LMICs.
Darolutamide is an oral second-generation androgen receptor antagonist approved by the US FDA in 2019. For the treatment of non-metastatic castration-resistant and metastatic castration-sensitive prostate cancer, clinical guidelines recommend androgen receptor antagonists, such as darolutamide, in combination with androgen-deprivation therapy as the standard of care. Unlike other second-generation androgen receptor antagonists, darolutamide has a distinct structure, which may offer advantages in tolerability and efficacy. With approval for both of these advanced prostate cancer types, darolutamide stands out as a key therapeutic option. Darolutamide primary patent has been granted in many LMICs and is expected to expire in May 2030. The patent term has been extended in few LMICs until 2035. Secondary patents have not been identified in LMICs.
A targeted combination of dabrafenib (a BRAF inhibitor) and trametinib (a MEK inhibitor) has shown significantly improved response rates, progression-free survival, and safety compared to traditional chemotherapy. Both drugs are FDA-approved for use in children, with age-appropriate formulations available , and each may also be used as monotherapy for other MAPK pathway-driven tumours. However, the use of these medicines requires BRAF mutation testing, which may limit access in LMICs. Of notice, these agents have a broader indication, such as for melanoma, and have a large use in adult diseases. The primary patents on dabrafenib and trametinib (owned by Novartis) expire in 2025 and 2029 respectively. Secondary patents on the combination extend to 2030, and additional formulation patents may last until 2033–2038. The primary dabrafenib patent is present in over 30 LMICs, with secondary patents on the combination in 25 LMICs. As part of the PADO–cancer process, the MPP was asked to explore licensing options for this combination.
In 2021, 537 million adults (10.5% of the global population) are grappling with diabetes. Projections indicate that this number will climb to 643 million by 2030. Approximately 240 million individuals worldwide live with undiagnosed diabetes. Notably, 90% of these undiagnosed cases are concentrated in LMICs. Type 1 diabetes (T1DM) affects over 1.2 million children and adolescents, with 54% of them under the age of 15. Type 2 diabetes (T2DM) is the most common type of diabetes, accounting for over 90% of all diabetes worldwide. Globally, the prevalence of type 2 diabetes is high and rising across all regions and has also become a concern in children and young people as a result of an increasing prevalence of obesity.
In 2022, one in eight people worldwide were living with obesity. Obesity and type 2 diabetes are closely linked, driving cardiometabolic conditions such as cardiovascular and kidney diseases, which present major health challenges, particularly in LMICs. By 2035, 79% of individuals living with obesity are expected to be in LMICs. Obesity trends and metabolic dysregulation are also rising among people living with HIV. With increased life expectancy, this population faces a growing burden of NCDs, including CVDs and diabetes. In South Africa, a recent study found that 63% of people living with HIV were overweight or obese, and 6% had diabetes. Ensuring accessibility and affordability of effective treatments is crucial, as the high cost of current medications poses significant barriers, particularly in LMICs with a high HIV burden.
More on MPP’s contribution in the diabetes field
Cardiovascular diseases (CVDs) stand as the leading global cause of death, accounting for 32% of all deaths in 2019, with an estimated 17.9 million lives lost. Over three-quarters of CVD deaths occur in low- and middle-income countries. These diseases were responsible for 38% of the 17 million premature deaths (those under 70 years old) attributed to noncommunicable diseases in 2019. Early detection of cardiovascular disease is paramount to initiate timely management through counselling and medication2. Despite high-quality scientific evidence of the benefits of different classes of drugs in preventing and controlling cardiovascular disease, their current use remains low.
The incretin hormones are gut hormones that amplify nutrient-induced insulin secretion in response to meal intake. Incretin peptides, principally Glucagon-like Peptide-1 (GLP-1) and Gastric inhibitory polypeptide (GIP), regulate islet hormone secretion, glucose concentrations, lipid metabolism, gut motility, appetite and body weight, and immune function, providing a scientific basis for utilizing incretin-based therapies in the treatment of type 2 diabetes1. Glucagon-like peptide-1 receptor agonists (GLP-1 RAs) are attractive options for the treatment of T2DM as they effectively reduce glycaemia and weight while posing a low risk of hypoglycaemia. Some GLP-1 RAs also have documented beneficial effects on the cardiovascular system, chronic kidney disease and non-alcoholic fatty liver disease.
Orforglipron is a once-daily oral, non-peptide (small-molecule) GLP-1 receptor agonist that can be taken at any time of day without food or water restrictions, originally discovered by Chugai Pharmaceutical and licensed by Eli Lilly. On 1 April 2026 the US FDA approved it for chronic weight management in adults with obesity or overweight with at least one weight-related comorbidity, making it the first small-molecule GLP-1 receptor agonist to reach the market. Its pivotal Phase 3 programmes (ATTAIN for obesity and ACHIEVE for type 2 diabetes) demonstrated significant weight loss, meaningful A1C reductions and improvements in cardiometabolic risk factors, with a safety profile broadly consistent with the injectable GLP-1 class, including a boxed warning for thyroid C-cell tumour risk. A regulatory submission for the type 2 diabetes indication is anticipated during 2026, and Lilly has filed for weight management and/or type 2 diabetes in more than 40 countries. Its oral formulation and fully synthetic, non-peptide nature offer potential advantages in manufacturing scalability and cost, which is particularly relevant for access in LMICs. The compound patents on orforglipron are granted in 32 LMICs and pending in a further 18 LMICs, and are expected to expire in 2037.
Tirzepatide is a once-weekly subcutaneous dual GIP and GLP-1 receptor agonist that is highly effective in achieving and maintaining glycaemic targets in people with type 2 diabetes and in promoting weight loss in adults with obesity or overweight. It is approved for type 2 diabetes and for chronic weight management, and has additionally been approved for the treatment of moderate-to-severe obstructive sleep apnoea in adults with obesity. Further trials are ongoing in chronic kidney disease and in cardiovascular and all-cause morbidity/mortality in obesity, alongside studies of major adverse cardiovascular events in type 2 diabetes. The primary patent on tirzepatide has been granted in at least 37 LMICs, including India, and is pending in a further 14, with an expected expiry in 2036. Secondary patents on the subcutaneous formulation have been filed and granted widely across LMICs and are expected to expire in 2039.
There are various approved formulations of semaglutide, to be administered orally or subcutaneously.
Oral semaglutide Oral semaglutide is a once-daily peptide-based GLP-1 receptor agonist that is highly effective in achieving and maintaining glycaemic targets while also promoting weight loss in people with type 2 diabetes. Its cardiovascular and weight-management indications have now been approved, having been anticipated filings at the time of earlier drafting. In October 2025 the FDA approved oral semaglutide to reduce the risk of major adverse cardiovascular events in adults with type 2 diabetes at high cardiovascular risk, covering both primary and secondary prevention, based on the SOUL trial. In December 2025 the FDA approved a higher-dose oral semaglutide 25 mg for chronic weight management and for reducing the risk of major adverse cardiovascular events in adults with overweight or obesity and established cardiovascular disease, making it the first oral GLP-1 receptor agonist approved for weight management, with US launch from early January 2026 and supported by the OASIS and SELECT trial programmes. The oral formulation is heat stable, which may make it particularly appropriate for use in resource-limited settings. Primary patents on semaglutide, expiring between 2024 and 2026, have been filed or granted in few LMICs. Secondary patents on semaglutide solid compositions with salcaprozate sodium (SNAC), an important excipient of the oral product, have been filed in about seventeen LMICs, including major manufacturing countries such as India, South Africa, China and Brazil, where they are expected to expire in 2031. Novo Nordisk owns many secondary patents that may extend exclusivity until 2040.
Subcutaneous semaglutide Subcutaneous semaglutide is a once-weekly peptide-based GLP-1 receptor agonist that is highly effective in achieving and maintaining glycaemic targets in people with type 2 diabetes. It also promotes weight loss in adults with overweight or obesity and reduces the risk of major adverse cardiovascular events in this population. Its therapeutic profile has since broadened considerably across several approved indications. In January 2025 it became the first GLP-1 receptor agonist approved to reduce the risk of worsening kidney disease and cardiovascular death in adults with type 2 diabetes and chronic kidney disease, based on the FLOW trial. In August 2025 it received accelerated FDA approval for the treatment of non-cirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with moderate-to-advanced fibrosis, becoming the first GLP-1 receptor agonist approved for MASH, alongside resmetirom as the only approved therapies for this indication, on the basis of the Phase 3 ESSENCE trial in which resolution of steatohepatitis without worsening of fibrosis was achieved in 63% of patients on semaglutide versus 34% on placebo at week 72. In heart failure with preserved ejection fraction (HFpEF) and obesity, the STEP-HFpEF programme demonstrated clinically meaningful improvements in heart-failure symptoms, physical limitations, exercise function and weight, alongside reduced inflammation; a regulatory submission for an HFpEF indication has been made on this basis, with a decision anticipated in 2026, though the indication is not yet approved. Taken together, semaglutide remains the GLP-1 receptor agonist with the most advanced and broadest clinical profile. Primary patents on semaglutide, expiring between 2024 and 2026, have been filed or granted in few LMICs, and patents on the subcutaneous formulation expired in 2024; however, Novo Nordisk owns many secondary patents that may extend exclusivity until 2040 in some jurisdictions.
Retatrutide (LY3437943) is an investigational once-weekly subcutaneous triple hormone receptor agonist, activating the GIP, GLP-1 and glucagon receptors, and is the first triple agonist to reach Phase 3 development. In the phase 3 TRIUMPH-1 obesity trial (2,339 adults with obesity or overweight without diabetes), the 12 mg dose produced an average weight loss of 28.3% at 80 weeks, among the largest reductions recorded in obesity pharmacotherapy, with around one third of participants reaching a healthy body weight (BMI ≤25), alongside improvements in knee osteoarthritis pain and moderate-to-severe obstructive sleep apnoea evaluated in parallel. In the phase 3 TRANSCEND-T2D-1 diabetes trial (537 adults with recent-onset type 2 diabetes), retatrutide reduced HbA1c by up to 2.0% and produced 16.8% weight loss at 40 weeks, with about 90% of participants reaching an HbA1c below 7%. Improvements in cardiovascular risk factors, including triglycerides and systolic blood pressure, were seen across both trials, and the safety profile was consistent with the incretin class, with predominantly mild-to-moderate gastrointestinal effects. A broad Phase 3 programme continues across type 2 diabetes (including a head-to-head trial against semaglutide), cardiovascular and renal outcomes, obstructive sleep apnoea, knee osteoarthritis and MASLD, with a regulatory filing anticipated in late 2026 or early 2027. The primary patent on retatrutide has been granted so far in at least 16 LMICs, including India, and is pending in a further 30 LMICs, with an expected expiry in 2038.
MPP is exploring ways to improve access to long-acting insulin analogues in LMICs, where the burden of diabetes poses a profound challenge, and appropriate treatment options remain limited and/or non accessible. Analogue formulations of insulin offer significant clinical advantages over traditional human insulins that are currently the most widely used insulins in LMICs. Their improved pharmacokinetic profiles support better glycaemic control, reduce the risk of hypoglycaemia and allow for more flexible dosing schedules . The clinical advantages of insulin analogues over human insulin led to their inclusion in the WHO Model List of Essential Medicines in 2021, recognising their potential to improve diabetes management globally. Despite these benefits, the high cost of insulin analogues continues to limit their uptake in LMICs, where human insulins remain more affordable and widely used. MPP’s engagement in this space seeks to address affordability challenges and contribute to broader access to newer, more effective diabetes treatments in underserved settings, while also monitoring progress in next-generation insulin formulations that offer sustained, longer-acting coverage with the potential to further reduce hypoglycaemia risk and ease the injection burden for people living with diabetes. Given the diversity within the class of insulin analogues, a snapshot card was not produced, as it would not adequately capture the specificities of the different products. While patents have expired for most insulin analogues (administered more often than weekly), technology transfer, including to strengthen regional manufacturing, could support an MPP intervention within this area.
Fixed-dose combination medicines for CVD, also known as polypills, have been shown to simplify treatment, improve adherence, and help manage key risk factors. These pills combine cholesterol-lowering medicines, blood pressure drugs, and, when appropriate, aspirin, to significantly reduce illness and death caused by atherosclerotic CVD. Their use is suggested for both primary and secondary prevention of CVD. The need for effective and accessible CVD prevention is especially important for people living with HIV, who face a higher risk of developing heart disease but have limited tailored prevention options. A recent study found that those who took a medicine that lowers cholesterol had fewer major heart problems than those who received a placebo over about five years, underscoring the importance of expanding prevention strategies in this group. The proven benefits and cost-effectiveness of fixed-dose combination therapies support their broader use and led to their inclusion on the WHO Essential Medicines List in 2023.
Cystic fibrosis is a rare, progressive, life-threatening disease, caused by mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene that results in the formation of thick mucus that builds up in the lungs, digestive tract, and other parts of the body. Cystic fibrosis leads to severe respiratory and digestive problems as well as other complications including diabetes, meningitis, osteomyelitis as well as skin and soft tissues infections. There is currently no cure for the condition and people with cystic fibrosis need daily treatments depending on the severity of their symptoms. There are no global annual deaths estimates for cystic fibrosis and related life expectancy, but it is generally assumed that in the absence of CFTR modulators, which remain not accessible in low- and middle-income countries, the median life expectancy for cystic fibrosis may be around 25 years (compared to 46 in presence of CFTR modulators).
The combination of the active substances elexacaftor, ivacaftor and tezacaftor is the first triple combination therapy available to treat patients with the most common cystic fibrosis mutation. It has been approved for patients 12 years and older with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is estimated to represent 90% of the cystic fibrosis population.
The combination of deutivacaftor, tezacaftor and vanzacaftor is a triple combination therapy of CFTR modulators, approved by US FDA and UK MHRA to treat patients 6 years and older, who have at least one F508del mutation in the CFTR gene or another responsive mutation, including 31 additional mutations. This broadens access for patients who previously had no approved treatment options. Compared with elexacaftor / tezacaftor / ivacaftor, it is taken as once daily tablet but has not yet been approved for children under six years old.
Sickle cell disease is a genetic disorder that affects haemoglobin, the protein responsible for transporting oxygen in red blood cells. The mutation that causes the disease causes the cells to become sickle-shaped, leading to blockages in blood flow, pain and organ damage. According to a study by the Global Burden of Disease, 7.74 million people were living with sickle cell disease in 2021. In the same year, more than 500,000 babies were born with the disease, more than three-quarters of them in sub-Saharan Africa. Sickle cell disease is the most common genetic disorder among people of African, Mediterranean, Middle Eastern and Indian descent. Early diagnosis of sickle cell disease is essential to ensure prompt treatment and control of the disease, as well as to prevent complications. Hydroxyurea is considered the most established disease-modifying treatment, but access to it remains difficult in low- and middle-income countries. This is why MPP works in particular with the WHO's Global Accelerator for Paediatric Formulations (GAP-f). At the same time, MPP has identified small molecule candidates in development that have a different mechanism of action from hydroxyurea and show promising potential. These could be added to the therapeutic toolbox.
Etavopivat is an oral, small-molecule activator of pyruvate kinase R (PKR) designed to increase red blood cell ATP production, thereby reducing the sickling of hemoglobin S-containing erythrocytes. Etavopivat demonstrated improvements in hemolytic markers (hemoglobin, bilirubin, reticulocyte count) and patient-reported outcomes in adults with sickle cell disease. Results of the phase III program HIBISCUS, etavopivat meet both co-primary substantially reducing vaso-occlusive crisis events and improving haemoglobin response in sickle cell disease. Novo Nordisk plans to submit for the first regulatory approval of etavopivat in the second half of 2026.
Mitapivat is an oral, small-molecule activator of pyruvate kinase R (PKR) designed to increase red blood cell ATP production, thereby reducing the sickling of hemoglobin S-containing erythrocytes. Mitapivat demonstrated promising anti-hemolytic agent in SCD, with clear efficacy in improving anemia and markers of hemolysis. The phase III RISE UP clinical trial did meet its primary endpoint of hemoglobin response, with mitapivat demonstrating a statistically significant improvement compared to placebo. While mitapivat also showed a reduction in the primary endpoint of annualized rate of sickle cell pain crises (SCPCs) compared to placebo, this trend did not achieve statistical significance.
NDec is an investigational oral, small-molecule fixed-dose combination of decitabine (a DNA methyltransferase 1 inhibitor) and tetrahydrouridine (a cytidine deaminase inhibitor), being developed by Novo Nordisk for sickle cell disease. By inhibiting DNMT1, decitabine reactivates fetal haemoglobin (HbF) expression, reducing HbS polymer formation and sickling, while tetrahydrouridine blocks the enzyme that otherwise rapidly degrades decitabine, enabling oral, non-cytotoxic dosing.
It is administered orally with food on a weight-banded, twice-weekly schedule, targeting a decitabine dose of approximately 0.16 to 0.25 mg/kg with tetrahydrouridine 8 to 12.5 mg/kg, and is currently being evaluated in the Phase 2 ASCENT1 trial. As an epigenetic HbF inducer, it occupies a distinct mechanistic class from PKR activators and, in principle, could be combined with them for additive or complementary disease modification.
Tebapivat (AG-946) is an investigational next-generation oral small molecule pyruvate kinase activator (PKR), structurally differentiated by its dual activation of the PKR and PKM2 isoforms expressed in red blood cells. It is engineered for greater potency and once-daily dosing. Currently in Phase 2 evaluation across hemolytic anemias including SCD and thalassemia, with early data suggesting a favorable tolerability profile and once-daily dosing potential. Topline data are expected for the second half of 2026.
Following its foundation in 2010, MPP initially focused on medicines for human immunodeficiency virus (HIV), with subsequent expansion to other infectious diseases, namely viral hepatitis and tuberculosis (TB) in 2015. This work led to licences that resulted in public health impact, as outlined in a MPP impact assessment modelling study published in 2022.
In 2018, MPP’s mandate expanded to target patented medicines included in the World Health Organisation (WHO) Model List of Essential Medicines (EML) or with potential for future inclusion, which encompass a whole range of disease areas, including cancers, diabetes and cardiovascular diseases, and is exploring other areas of intervention, as relevant. Additionally, MPP contributed to the COVID-19 response and its current mandate includes pandemic preparedness and response.
MPP’s strategy for the 2023-2025 period embraces a disease agnostic approach, by which patented medicines for which an MPP intervention would potentially make a difference in public health, might be considered for public-health oriented agreements with innovators, regardless of the health area.
MPP’s work started with small molecules, and after conducting a feasibility study on expanding access to biotherapeutics in 2022, expanded its mandate to biologics. Moreover, given their ground-breaking potential impact, long-acting technologies and formulations designed to achieve longer exposure to medicines are considered for prioritisation since 2021, together with any relevant medical technologies for which an MPP intervention might generate positive impact for public health.
In line with this strategy, MPP might consider for prioritisation candidates at any stage of development, from pre-clinical to marketed.
The latest MPP Prioritisation Report (2024) can be accessed through this link. To read previous prioritisation reports, follow this link.
It should be noted that MPP does not include in its prioritisation list, the medicines and health innovations for which it has already signed agreements. The list of MPP previously signed agreements to enable access are available on the “agreements with innovators” section of MPP’s website.
The MPP prioritisation framework was designed to answer the following three questions, as guiding principles. By addressing these questions, MPP collects insights about public health and access dimensions of the products assessed, as well as insights to assess the potential impact of an MPP intervention.
In order to guide products’ assessments, the framework addresses the following considerations, as guiding principles
1. Does the product address a public health need?
This question is assessed through the public health pillar of the framework, where the burden of the health condition is assessed, as well as the advantages of the candidate product over existing alternatives of care for this condition.
2. Are there any access hurdles (anticipated or existing) for the product in low-and-middle-income countries (LMICs)?
This question is assessed through the access pillar. It includes access considerations on which MPP directly intervenes through licensing and technology transfer (e.g., intellectual property, including patents and know how), as well as additional access considerations which may be important in the treatment cascade (e.g. access to diagnostics, access companion treatments or availability of health system enablers).
3. What would be the effect of an MPP intervention on access?
This question elaborates on both public health and access pillars, and ensures that medicines are prioritised where an MPP intervention could yield substantial health (and sometimes economic) impact.
Download
By addressing these questions, MPP collects insights about public health and access dimensions of the products assessed, as well as insights to assess the potential impact of an MPP intervention.
The framework is structured into seven pillars:
The assessment of medicines for licensing is carried out in collaboration with MPP’s Scientific Advisory Panel and Community Advisory Panel.
MPP’s prioritisation process generates two lists – a priority list and a watchlist – of medicines and health technologies for which expanded access in LMICs could provide significant health benefits over standards of care, and where a voluntary agreement, including licensing and/or technology transfer, through MPP could lead to substantial public health impact. These lists guide MPP in-licensing efforts.
Medicines and health innovations for which voluntary licensing and/or technology transfer through MPP would lead to expanded access, significant health benefits, and substantial public health impact compared to standards of care.
Medicines and health innovations for which expanded access could provide significant health benefits but for which supporting data are lacking and/or key challenges need to be addressed for expanded access through MPP licensing to lead to public health impact.
Additionally, medicines are sometimes provisionally added to the watchlist when a potential added benefit might be obtained through an MPP licence, but where a full assessment is still ongoing.
MPP is strongly committed to increase access to safe, effective and adapted products to support children’s health regardless of where they live. To illustrate this commitment, several products on the MPP prioritisation list for in-licensing and clearing the prioritization framework are tagged as “paediatric.”
If you would like to know more about MPP, its work on medicines prioritisation or want to collaborate, please contact science@medicinespatentpool.org