Breakthroughs Blog: FDA

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.  

A team of researchers from the United Kingdom, the United States, and South Africa is developing a vaccine for hantavirus, the virus behind a worrying outbreak on a cruise ship in the Atlantic Ocean. Hantavirus is typically spread by rodents, and transmission between humans is very rare. There is no specific treatment of vaccine for hantavirus, which, as seen in the current outbreak, can be deadly. The researchers are using an innovative method called insilication to make the vaccine thermally stable, aiming to reduce the high resource and economic costs of meeting the cold chain and storage requirements that are associated with many vaccines. Moderna’s mRNA-based seasonal flu vaccine outperformed conventional flu shots in a late-stage international trial of more than 40,000 adults aged 50 and older. The US Food and Drug Administration is currently reviewing the vaccine after rejecting the initial application and is expected to make a decision by August 5. If approved, the vaccine would be the first mRNA seasonal flu shut in the United States. mRNA vaccines can be produced much more quickly than conventional flu shots, which could enable public health authorities to wait longer before choosing which flu strains to target, giving them more time to gather up-to-date data about circulating strains before flu season begins. A research team has discovered a previously unknown mechanism by which HIV-1 can infect resting cells, challenging previous assumptions and raising new possibilities for the development of improved treatments. For HIV to infect immune cells, it must deliver its genetic material into the cell’s nucleus, which is surrounded by a protective structure—how the virus has broken through the barrier has long puzzled scientists. Another obstacle to an HIV cure has been the latent reservoir of resting immune cells that house dormant virus, which cannot be cleared by treatment. The researchers found that when the virus spreads between immune cells, it triggers a signal that unlocks this protective gateway around the nucleus, allowing the virus to enter. They also found that this process does not require the immune cell to be activated, meaning that the reservoir can be established and maintained while cells are resting, upending long-standing assumptions and unlocking new avenues toward the development of a treatment that can destroy the virus and target the latent reservoir. 

2025 has been a whirlwind year for global health. In the United States, we've seen the integration of the US Agency for International Development (USAID) into the Department of State, transformation at the Department of Health and Human Services (HHS), and a complicated and prolonged budget process. Globally, we've seen setbacks and progress in HIV and malaria, an evolution at the World Health Organization (WHO), a multilateral win with the Pandemic Agreement adoption, and African partners stepping up to boost autonomy.As the year draws to a close, GHTC is reflecting on the innovations, policy shifts, and pivotal moments that defined 2025 and what they signal for the years to come.US global health leadership in transitionOn day one of his second term, President Donald Trump initiated an effort to realign US foreign assistance programs with the goal of making America safer, stronger, and more prosperous. This effort started with an overhaul of USAID, triggering a series of layoffs, stop-work orders that turned into award terminations, and withdrawals from multilateral partnerships, culminating in the agency’s official integration into the State Department on July 1.While this suspension and termination of USAID programs disrupted critical research projects and health services worldwide earlier this year, in September, the State Department unveiled the America First Global Health Strategy, which outlines a plan to reshape the US government’s global health programming, presenting opportunities for global health research and development (R&D).The new strategy centers multiyear bilateral agreements with countries while reducing ties with nongovernmental organizations and narrowing health focal areas. While critical questions remain on implementation—including on whether the agency still retains needed expertise and how it will coordinate with interagency technical partners like the US Centers for Disease Control and Prevention (CDC)—the strategy’s focus on integrating aid more directly into national health systems is a step forward in improving sustainability and self-reliance. The strategy also raises the exciting possibility that funds could be used to support “innovative, breakthrough technologies.”  Building on USAID’s successes and shortcomings in that space, the department has a clear opportunity to establish a centralized R&D fund—in complement to bilateral agreements—to invest in innovative tools that could leapfrog global health progress. Through collaboration with global health stakeholders and interagency partners, State could reshape US global health programs to continue to save lives; foster funding beyond US government resources, including through partner nations; and promote shared security and prosperity.Overhaul of US scientific and public health infrastructureThe United States has long been a leader in global health R&D and science more broadly, with American researchers, universities, companies, and patients benefiting. In 2025, sweeping changes at HHS reshaped America’s public health and research infrastructure. These far-reaching reforms aimed to advance administration priorities of promoting “gold standard science,” improving efficiency, transparency, and oversight and realigning investments to match administration objectives. At the National Institutes of Health (NIH) and CDC, these changes have included significant staff departures, among them seasoned leaders, and the appointment of many officials who were previously critics of these agencies. It has also included new policies to overhaul oversight of foreign awards and cap indirect costs, changes to longstanding vaccine guidance, and the terminations of numerous awards, including a wind-down of mRNA vaccine development activities. The speed and scale of these changes have raised concerns across the biomedical research and public health communities. While thoughtful reforms could yield benefits, experts warn that current actions risk driving US scientists abroad, disrupting promising clinical trials, weakening the pipeline of new tools, eroding trust in public health guidance, and weakening the structures that have sustained US scientific leadership. US budget swings and shutdowns2025 began with a race to fund the government, with President Trump signing the fiscal year 2025 budget into law in mid-March. While the budget maintained global health R&D-relevant funding lines at prior levels, it funded a government that now looked significantly different after sweeping layoffs and restructuring. Months later, the administration issued its budget request, proposing significant cuts and a restructuring of global health and research programs, reflecting far-reaching changes it had already begun to enact.In June, the administration proposed a request to rescind billions in funding for foreign aid. Though Senators stepped in to remove cuts to global programs for HIV/AIDS and several other health areas, the package ultimately became law, cancelling $500 million in previously appropriated global health funds and raising the possibility that this administration will continue to use rescissions as a tool to withdraw funding.  By late summer, Senate and House appropriators advanced interim funding bills that included higher-than-expected funding for global health and medical research, maintaining funding proposed for elimination by the administration. But then stalled negotiations triggered the longest-ever government shutdown, during which the administration implemented unprecedented furloughs and layoffs that affected critical staff at public health and health research agencies.While the shutdown ended on November 12, pushing the funding fight until January, and most associated layoffs were reversed, uncertainty about future funding for global health R&D persists, especially with a deeply divided Congress and the potential of further recissions.A year of setbacks and breakthroughs in HIV and malaria effortsGlobal HIV/AIDS and malaria efforts faced major setbacks in 2025 as declining US and international donor support has threatened decades of hard-fought progress. Funding cuts could lead to millions of additional HIV and malaria cases in the coming years and has already stalled promising research advancing new HIV and malaria tools.Amid these challenges, the June approval by the US Food and Drug Administration of lenacapavir, a twice-yearly injectable drug for HIV prevention, marked a major milestone and a reminder of the incredible progress possible through strong support for research. Developed by Gilead and supported by NIH-backed research, the drug offers an alternative to daily pills or bi-monthly injections, making prevention more accessible and easier to maintain. Global partners are stepping up to expand access, and Phase 3 trials of a once-yearly version are planned.In November, Medicines for Malaria Venture and Novartis shared Phase 3 trial results for GanLum, a new drug found to be highly effective at treating malaria, including drug-resistant cases, and capable of blocking disease transmission. Pending approval, it could help counter rising resistance to current treatments.Other notable R&D advances in 2025 also targeted the growing threat of antimicrobial resistance, including a Phase 3 trial for a novel antibiotic targeting a dangerous antibiotic-resistant pathogen, support for what could be the first-ever vaccine for Escherichia coli, and efforts to harness artificial intelligence to transform antibiotic discovery. These breakthroughs underscore the importance of sustained R&D investment and the efforts to ensure new tools reach those who need them.WHO restructures amid budget shortfallFacing a severe budget crisis and pressure from member states to reprioritize programs, WHO launched a sweeping overhaul in 2025, cutting staff, slashing its budget, and reorganizing core functions. At its February Executive Board meeting, member states approved a $1.1 billion budget reduction, leaving a remaining $1.7 billion shortfall for 2026 and 2027, despite a recent increase to membership dues and appeals for voluntary contributions.In late April, WHO announced a plan to halve its senior leadership team and consolidate departments. Infectious disease and climate and health programs remain relatively well-resourced, and merging several disease-specific initiatives into a new division could strengthen certain programs. However, neglected tropical diseases and health emergency preparedness and response programs face sharp cuts, while reductions in regional and in-house expert posts may weaken global lab and surveillance networks and delay critical guidance, raising concerns about WHO’s outbreak response capacity.One core R&D function spared from the worst cuts was WHO’s prequalification (PQ) program, which evaluates medical products to support regulatory approval and rollout in countries with less advanced regulatory authorities. The program has marked some important achievements this year, launching a new portal to improve transparency for developers, introducing a new streamlined review and guidance process for TB diagnostics, and prequalifying lenacapavir for HIV prevention through a compressed process completed in just 36 days. As WHO and the broader United Nations system grapple with financial strain and diminished donor support, questions remain about the capacity to deliver on their mandate. A major outbreak could test the limits of this leaner system.Pandemic Agreement provides hope in challenging year for multilateralismAt the World Health Assembly in May, delegates adopted the Pandemic Agreement, a milestone win for global health after three years of complex negotiations. However, consensus on a pathogen and access-benefit sharing system was not reached, requiring member states to return to the negotiating table to finalize an annex for adoption at next year’s assembly. Persistent disputes over the voluntary or legally binding nature of participation signal tough negotiations ahead.While the Pandemic Agreement is a huge win, pushback from key countries and declining pandemic preparedness investments raise concerns about global readiness. Annex negotiations and the 2026 United Nations High-Level Meeting on Pandemic Prevention, Preparedness, and Response will further test international unity on this crucial topic.Meanwhile, 2026 brought notable R&D advances to strengthen our defenses against pandemic threats. The Coalition for Epidemic Preparedness Innovations launched the first-ever adjuvant library of 25 immune-boosting substances to improve vaccine effectiveness and awarded $17.3 million for Phase 2 trials of a promising Nipah vaccine, among the first candidates targeting the priority virus to reach this critical stage. IAVI’s vaccine candidate for Lassa virus, another virus with pandemic potential, was also found to be safe and effective in a first-in-human Phase 1 trial, marking further R&D progress against concerning disease threats.Partners step up to fill gaps and boost autonomyAmid steep foreign aid funding losses, African nations are accelerating efforts to boost manufacturing and laboratory capacity on the continent and reduce reliance on external partners. At October’s Conference on Public Health in Africa in Durban, South Africa, thousands of participants endorsed The Durban Promise, a roadmap for Africa’s health sovereignty, urging action on manufacturing, financing, workforce and capacity development, and regulatory strengthening, a signal of growing continental unity.This momentum was echoed by the Accra Reset, led by Ghanaian President John Mahama, to forge further progress toward greater sovereignty and more equitable global cooperation. At the continental level, the Africa Centres for Disease Control and Prevention launched a new biomanufacturing fellowship to help meet the goal of producing 60 percent of Africa’s vaccine dose needs locally by 2040.South Africa held the G20 presidency this year, culminating in the first G20 summit held on African soil and a declaration focused on priorities for Africa and the Global South. The summit was punctuated by the absence of the United States, underscoring the imperative for greater African autonomy as international aid declines and health threats rise. With the United States assuming the G20 presidency next year, its priorities will be closely watched. Looking ahead to 2026If 2025 has taught us anything, it’s that global health is unpredictable and constantly evolving, so we won’t speculate on exactly what 2026 may bring. But a truth remains clear: global health R&D is a strong investment that pays off—saving lives, strengthening economies, and fueling scientific progress. As the United States redefines its approach and role in the broader global health ecosystem, it remains to be seen how it, and others, will step up to ensure that promising technologies in the pipeline are advanced to improve lives around the world.For other fascinating global health news delivered to your inbox weekly and to stay abreast of developments in 2026 and beyond, sign up now to receive GHTC’s R&D News Roundup newsletter.

Last Wednesday, Emergent BioSolutions announced that it has received a $56 million contract extension with the Administration for Strategic Preparedness and Response to supply the US government with doses of a combined smallpox/mpox vaccine. The single-dose vaccine was first approved by the US Food and Drug Administration in 2007 for smallpox and, since 2024, has been one of two approved vaccines for smallpox and mpox. The vaccine doses, which will be stored in the Strategic National Stockpile, aim to boost US preparedness for future public health emergencies. A novel breakthrough method that utilizes artificial intelligence and advanced imaging technologies to more accurately and efficiently identify therapeutic antibodies could transform the way researchers develop treatments for infectious diseases, particularly emerging diseases, by significantly accelerating research. The method reduces the time needed to identify antibodies with potential from weeks to under a day and can also accomplish this task more efficiently, requiring far fewer researchers dedicating their time to screening thousands of candidate antibodies. This could allow researchers to move on from antibody discovery to the later stages of drug and vaccine development earlier and, ultimately, bring lifesaving products to patients faster. A recently published study found promising results for a new approach to developing broad-spectrum antivirals to treat existing viral infections and maybe even to prevent infections from emerging viral threats. Broad-spectrum antivirals are treatments that could combat a wide range of viruses by hitting essential targets that are common across many different viruses. While there are broad-spectrum treatments for bacterial infections, there has not been a similar broad-spectrum antiviral treatment approved for human use, in part because the proteins on the surface of a virus vary widely and change rapidly over time. The researchers focused on small molecules called synthetic carbohydrate receptors (SRCs), which earlier studies have suggested could be a potential target for antiviral medicines. They tested 57 different SRCs against six deadly viruses from three different viral families, including the viruses behind COVID-19, MERS, Nipah, and Ebola, finding several promising SRCs that blocked cells in the lab from being affected by all the viruses, some of which also later demonstrated promising safety and efficacy results in mice.