Breakthroughs Blog

The Democratic Republic of the Congo has approved the use of a new single-dose oral treatment for sleeping sickness, a potentially fatal disease which is endemic in several countries in Central and West Africa. The approval of Acoziborole Winthrop, codeveloped by the Drugs for Neglected Diseases initiative (DNDi) and Sanofi, follows successful Phase 2 and 3 clinical trials conducted in the DRC and Guinea by DNDi in partnership with national authorities, which demonstrated that the drug is safe and effective. Existing treatments require a ten-day oral treatment regimen or a combination of injection and oral therapy that requires several days of hospitalization for patients in the most advanced stages. The DRC approval, which will make the drug available free of cost to patients in the country, is expected to be followed by endorsements in other endemic countries soon.A research team from the University of Maryland School of Medicine’s Center for Vaccine Development and Global Health has shared promising early results from a first-in-human clinical trial comparing a new investigational vaccine against Lassa fever and rabies to a licensed rabies vaccine, finding that the vaccine candidate was safe and induced rapid and robust immune responses against both viruses. There are currently no available vaccines for Lassa fever, a dangerous disease considered a priority threat with epidemic and pandemic potential that is common in parts of Western and sub-Saharan Africa, which also have a high burden of another severe disease: rabies. A dual vaccine could help streamline health care delivery in settings with limited health care infrastructure; the vaccine candidate can also be freeze-dried for storage, making it well suited to low-resource settings where it can be difficult to maintain the cold chain. Last week, DNDi, the Medicines for Malaria Venture (MMV), and the Global Antibiotic Research and Development Partnership (GARDP) announced a new cooperative agreement to explore how the three organizations can work together to advance global health research and development (R&D) amid global aid cuts and calls to reshape the global health architecture. The organizations plan to pool their expertise and resources to advance the R&D of effective and affordable medicines, while centering patient needs and equity. In addition, the partners will collaborate on policy advocacy and communications activities to raise awareness of the impact and promise of the nonprofit product development partnership model in advancing innovative, lifesaving medicines for critical, but often overlooked, diseases.  

Every May for the last 79 years, member states of the World Health Organization (WHO) have gathered in Geneva, Switzerland for the annual World Health Assembly (WHA). The Assembly remains one of the most important moments on the global health calendar, offering insight into how countries are responding to emerging health challenges and where political momentum is building around the future of global health governance, financing, and innovation. This year, however, Geneva felt markedly different. Conversations throughout the week reflected an ecosystem in transition. The global health community is grappling with constrained resources and evolving geopolitical priorities. Meanwhile, WHO reform efforts, persistent budget challenges, and upcoming leadership transitions across multilateral institutions contributed to a broader sense that global health is entering a new phase. Adding to this uncertainty, the United States did not formally participate in assembly proceedings for the first time in WHO's history following its withdrawal from the organization earlier this year.  The assembly also convened amid an evolving Ebola emergency and recent hantavirus outbreak, grounding at times abstract debates about preparedness, coordination, and health security in the realities of active disease outbreaks affecting communities around the world. Against this backdrop, WHA served not only as a decision-making forum but also as a barometer of where global health is headed. For GHTC, three high-level themes emerged that will help shape our advocacy in the months ahead:  1. Innovation must be matched by scale, delivery, and sustainable financing.  Throughout the week, one message surfaced consistently across discussions with WHO leaders, product developers, donors, and civil society organizations: scientific breakthroughs are essential to improving health outcomes, but their impact depends on whether new tools can be produced, financed, delivered, and accessed by the communities that need them most. Today, the challenge is twofold. Continued investment in R&D remains critical to delivering the next generation of vaccines, diagnostics, and therapeutics, particularly for diseases and conditions where significant gaps persist. At the same time, many effective technologies already exist but are not reaching those who need them. Addressing both realities will require increased financing across the R&D to delivery continuum, accelerated regulatory pathways, increased local manufacturing capacity, and more responsive procurement and delivery systems to ensure that innovations are both advanced and translated into impact.  This dual imperative carries particular urgency amid ongoing uncertainty in global health financing. As donors reassess priorities and international institutions face budget constraints, important questions remain about who will finance the next generation of health innovations and ensure their deployment at scale.  Discussions throughout WHA highlighted growing concerns that funding across the entire innovation continuum, from R&D to product introduction, market shaping, and implementation, may not keep pace with scientific advances and need, widening the gap between what is possible and what is accessible. 2. Country ownership is operational, not aspirational.  Perhaps the most significant shift visible throughout Geneva was the growing expectation that countries and regions will increasingly drive and implement their own health priorities.This was particularly evident in discussions marking the first anniversary of the Global Coalition for Local and Regional Production, Innovation, and Equitable Access. Representatives from France, the United Kingdom, the European Union, Indonesia, South Africa, and the African Union repeatedly emphasized the coalition's goal of enabling countries—particularly in the Global South—to become producers, developers, and innovators of health technologies. This conversation reflected a fundamental evolution in how many stakeholders view health security and access: local and regional production is no longer seen primarily as a pandemic preparedness objective but rather as a cornerstone of resilient health systems, economic development, and long-term sustainability. At the same time, discussions throughout the week underscored that manufacturing alone is insufficient; strong regulatory systems are equally critical to ensuring that health products reach communities efficiently and safely. This was reinforced by WHO's newly announced partnership with the African Medicines Agency (AMA) aimed at strengthening regulatory harmonization and reliance, improving safety surveillance, building regulatory capacity, and supporting innovation and local manufacturing across the continent. Similar themes also emerged from GHTC's dialogue with WHO's Prequalification (PQ) team. WHO officials described PQ as an indispensable but ultimately transitional program. The organization's long-term vision is for mature national and regional regulatory authorities to reduce reliance on PQ over time. For that vision to become reality, institutions like AMA and other regional regulatory bodies must develop the technical capacity, regulatory maturity, and trust needed to independently oversee health products.3. AI and emerging technologies are already being deployed.  During WHA week, it became clear that artificial intelligence (AI) and other emerging technologies are no longer hypothetical future solutions, they are already being integrated into health systems and product development. The focus has shifted from how they could be used to how they can be deployed and governed more effectively.  For instance, within WHO's PQ program, the PQ team highlighted the growing use of digital tools and AI-enabled systems to improve efficiency and transparency. Earlier this year, WHO launched its electronic prequalification submission portal, now the mandatory application submission pathway for manufacturers. Looking ahead, the PQ team is exploring how AI tools can streamline product reviews and reduce administrative burdens. Beyond regulatory systems, member states and stakeholders debated WHO's broader role in shaping governance frameworks for AI and digital health as part of the official WHA agenda. While many emphasized AI's potential to boost productivity, discussions consistently returned to concerns of how the organization will navigate challenges in transparency, accountability, data privacy, and public trust.Another major recurring concern was the risk that technological advances could deepen existing disparities if lower-resource countries lack the infrastructure, expertise, or regulatory frameworks necessary to benefit from them. As AI becomes increasingly integrated into global health R&D and delivery, ensuring that these technologies narrow, rather than widen, health gaps will be an increasingly important challenge.Carrying these lessons forward WHA79 underscored that global health is entering a new chapter, one defined by financial constraints, shifting political dynamics, evolving models of partnership, and rapid technological change. Yet amid this uncertainty, one message remained clear: the pursuit of equitable access to health innovation is both a global priority and a shared responsibility. As a coalition, GHTC will carry three priorities forward from Geneva: 1. We will continue advocating for innovation across the full product lifecycle. Investments in research, development, and access pathways must occur in tandem, so new technologies can reach the communities they are intended to serve at scale. For GHTC, this reinforces the importance of maintaining a diverse ecosystem of product developers, manufacturers, implementers, procurement agencies, regulators, civil society organizations, and community leaders to transform scientific advances into public health gains.  2. We recognize the growing emphasis on country ownership and the value of local leadership. As governments increasingly prioritize local production and health sovereignty, GHTC will work to ensure that R&D is a core pillar of these efforts through our Global Partners Network, dialogues with WHO, and engagement in global forums.  3. We will advocate for a future in which all countries have the capacity to contribute to and see their people benefit from the development of lifesaving technologies. Emerging technologies hold tremendous promise for advancing R&D against global health threats. Realizing that promise, however, will require intentional efforts to accelerate R&D, strengthen local capabilities, expand access, build trust, and promote equitable governance. GHTC will support policies that bolster innovation ecosystems worldwide, not only in countries with the greatest resources.The conversations in Geneva made clear that the future of global health will be shaped by parallel progress in innovation and access—driving new breakthroughs while strengthening the systems and local leadership needed to deliver them equitably at scale to the people who need them most. 

A Phase 1 clinical trial testing a candidate that could become the first vaccine for leishmaniasis, a disfiguring skin disease, is set to begin soon in Kenya and Brazil, where the disease in endemic, as well as in Cincinnati, Ohio, as the disease risk grows in the United States. The Global Health Innovative Technology (GHIT) Fund is supporting the trial, which will test the safety and immune response of the candidate in healthy people. The vaccine, which was developed using CRISPR gene-editing technology to mutate the parasite that causes the disease, previously demonstrated strong results in mice and has received US Food and Drug Administration approval as an investigational new drug, which allows the product to be tested in humans. The Coalition for Epidemic Preparedness Innovations (CEPI) has announced that it is providing funding to fast-track the development of three Ebola Bundibugyo virus vaccine candidates, as the concerning outbreak of the Bundibugyo strain, for which there are no approved vaccines, continues to grow in the Democratic Republic of the Congo and Uganda. Specifically, the funding will support preclinical development and Phase 1 clinical testing of Moderna’s mRNA vaccine candidate; help accomplish a key early step in vaccine manufacturing for IAVI’s vaccine, which is based on the organization’s rVSV vaccine platform; and help the University of Oxford prepare for a Phase 1 trial of its vaccine, which is based on the same platform as its Oxford/AstraZeneca COVID-19 vaccine.  Last week, Zendal announced the launch of the international MTBVAC AVANTE consortium to accelerate clinical development and prepare regulatory and policy pathways to ensure broad and equitable access of the MTBVAC tuberculosis (TB) vaccine candidate over the next four years. The current TB vaccine is more than 100 years old and offers inconsistent protection in adults against pulmonary TB, so a new and improved vaccine that is safe and effective across diverse populations who face TB risk will be essential to ending the pandemic. With funding from the Global Health EDCTP3, the consortium of 12 European and African partners will initially support two major ongoing trials in high-TB regions in sub-Saharan Africa, one evaluating the vaccine in adolescents and adults and one comparing the candidate’s protection in newborns to the existing vaccine.  

Amid the ongoing outbreak of the Bundibugyo strain of Ebola—for which there are no specific approved vaccines or therapies—in the Democratic Republic of the Congo and Uganda, the World Health Organization (WHO) has collaborated with outside experts to identify priority Ebola treatments and vaccines for clinical trials. On the treatment side, WHO recommended prioritizing Mapp Biopharmaceutical’s MBP134, Regeneron’s maftivimab, and Gilead Sciences’ antiviral remdesivir. For prevention, Gilead's experimental oral antiviral obeldesivir was highlighted as a priority for post-exposure use in contacts of confirmed cases. IAVI’s rVSV Bundibugyo was identified as the most promising vaccine candidate, but Oxford University and the Serum Institute of India’s ChAdOx1 Bundibugyo vaccine candidate could be available for testing sooner following additional animal testing. WHO is now working with authorities in Congo and Uganda and other partners to design and implement ethical clinical trials during the outbreak. An experimental drug for chronic hepatitis B could potentially serve as a functional cure for the virus, as demonstrated in recently published results from two international studies. For many, hepatitis B only causes short-term illness, but for some, it can become a chronic infection, gradually damaging the liver and, without treatment, leading to liver cancer, liver failure, or even death—it causes around 1.1 million deaths globally each year. Chronic infection requires lifelong therapy in the form of daily pills to reduce viral levels and prevent liver damage, which can be hard to adhere to or access in some countries. Bepirovirsen, developed by GSK and Ionis Pharmaceuticals, was able to reduce the virus in around one in five patients to levels low enough for the immune system to keep it under control without treatment. The drug is under fast-track review by the US Food and Drug Administration, which is expected to make a decision later this year, and is also being considered by international regulators in Japan, China, and Europe. A research team at Georgia State University has developed a promising antiviral drug candidate that could help treat measles, croup, and related viral diseases that cause contagious and life-threatening respiratory infections, as falling coverage of the vaccine for measles, mumps, and rubella raises the risk of infection. The drug, GHP-88310, was designed to be a once-a-day oral pill for vulnerable patent groups, like children and immunocompromised patients, which make up the majority of croup and measles cases. GHP-88310 has been shown to be well-tolerated in rats, mice, ferrets, and dogs at higher concentrations.