Breakthroughs Blog: Medicines

Newly published data shows that the antiviral compound mosnodenvir, developed by Johnson & Johnson, is effective at preventing dengue infection in people deliberately exposed to the virus, which affects millions around the world every year. There are no approved treatments for dengue, and available vaccines face production and efficacy issues. Despite its promise, the future development of the drug is in flux after Johnson & Johnson halted all its infectious disease work last year. Negotiations are underway to find another company to advance the drug, which, if it continues to demonstrate potential, could be used to protect large populations during epidemics, as well as to protect people traveling to endemic areas. Last week, Brazil’s regulatory agency approved the Butantan Institute’s Butantan-DV vaccine for dengue for people aged 12 to 59. The vaccine demonstrated high efficacy in clinical trials against dengue, which causes millions of cases globally, with a substantial burden faced by Brazil. The single-dose vaccine could offer faster and simpler vaccination campaigns compared to existing vaccines. Brazil signed an agreement with WuXi Biologics to deliver approximately 30 million doses of the vaccine in the second half of next year. The results of a human-challenge Phase 2a trial published last week found that an mRNA vaccine was more effective against symptomatic and febrile flu compared to the conventional vaccine. Another trial published last week also found that mRNA flu vaccines were more effective than conventional vaccines. Beyond effectiveness, mRNA vaccines can also be made more quickly, helping ensure that vaccines match circulating flu strains. Additional studies that include older people, those with coexisting illnesses, and school-age children, who face elevated risks, will be required to ensure broad efficacy across populations. 

At the recent Union World Conference on Lung Health, TB Alliance presented the results of a Phase 2 clinical trial showing that the novel antibiotic candidate sorfequiline has the potential to improve tuberculosis (TB) treatment when combined with pretomanid and linezolid in people with drug-sensitive TB. The regimen was more effective than the current standard of care, and sorfequiline also demonstrated greater activity than bedaquiline, a key component of the BPaL regimen, a previous breakthrough that drastically reduced treatment time for most drug-resistant TB. This new treatment could shorten TB therapy even further. TB Alliance is working with partners ahead of the launch of a Phase 3 trial next year. Researchers at the University of California San Diego and partners have developed a new method to combat antibiotic-resistant bacteria, which could pave the way for developing alternatives to traditional antibiotics for treating a wide range of deadly infections, as the global threat of antimicrobial resistance continues to grow. The researchers used bacteriophages (or phages), which are viruses that infect and kill bacteria. Phages have been used to treat bacterial infections for more than 100 years but are usually limited in the bacterial strains they can successfully target. Researchers overcame this by allowing the pages to evolve alongside bacteria in a controlled laboratory setting for 30 days, enabling the phages to adapt their bacterial defenses, becoming more effective at killing a wide variety of bacterial strains, including drug-resistant ones, and better at suppressing bacterial growth for extended periods. Researchers at Indiana University have developed a molecular test that can simultaneously detect three dangerous fungi much faster than traditional methods, helping patients receive accurate, timely treatment against this growing global challenge. The test works by targeting genetic regions unique to each fungus, and it was much faster and more accurate than standard methods, a key advance as fungal infections are often mistaken for other respiratory illnesses. While the test still needs to be clinically validated in further studies, the research team believe it could eventually fill a major gap in fungal diagnostics. 

It’s been a difficult year for people worldwide who live with, are at risk of, or research HIV. Funding reductions and shifting priorities among key donors have disrupted or halted critical research and health services. Around the world, major international donors have reduced their funding for global HIV programming, leading to stoppages and cutbacks of lifesaving care. In the United States, new policies and grant terminations at the Department of Health and Human Services have significantly disrupted HIV research. The integration of the US Agency for International Development into the Department of State and resulting award terminations also interrupted HIV research and service delivery globally.These reductions risk undermining hard-won global progress and could lead to a resurgence in cases. Already, we’re seeing declines in HIV testing and treatment initiation this year. This is especially disheartening given exciting progress in recent years: at the end of 2024, new HIV infections were down 40 percent and AIDS-related deaths down 50 percent since 2010. The last few years have also brought exciting research advances, building momentum toward breakthroughs that could change the course of the global pandemic.With science yielding fruit and new donor countries and private funders stepping up, there is still hope that through renewed partnership and investment, we can regain momentum toward ending HIV/AIDS as a global public health threat. Here are some of the year’s most exciting advances in HIV research and innovation this year that inspire optimism amid significant challenges.A breakthrough in HIV prevention and accessIn June 2025, the US Food and Drug Administration approved lenacapavir as the first twice-yearly injectable drug for HIV prevention, marking a major milestone in the fight against the virus, While the drug, developed by Gilead using evidence from NIH-funded research, was previously approved for treating multidrug-resistant HIV, the new indication offers a discrete, twice-annual alternative to daily pills or bi-monthly injections, making prevention more accessible and easier to maintain. The approval followed highly successful clinical trials, which were notable not only for how effective the drug was across key populations, but also for their inclusion of historically underserved populations.Following the FDA’s decision, Gilead and the Global Fund to Fight AIDS, Tuberculosis and Malaria announced an access agreement that will see Gilead supply doses, at no profit, for up to two million people over three years. The Gates Foundation and Indian manufacturer Hetero Labs are also partnering to manufacture generic lenacapavir at an affordable price. The World Health Organization swiftly recommended the drug’s use, and South Africa’s regulatory authority approved it at record speed, the first such approval in Africa. It’s exciting to see partners work together to help ensure this breakthrough can be accessible in the communities where it is most needed quickly and affordably, a sharp divergence from past HIV product rollouts that saw years-long delays before people in low- and middle-income countries received access to new tools. Earlier this year, Gilead also published data suggesting a once-yearly version of the drug may be possible, with Phase 3 trials to begin later this year.Lenacapavir is not the only exciting advance in long-acting HIV prevention we saw in 2025. Merck’s investigational once-monthly oral prevention pill was found to be well tolerated in adults in a Phase 2 trial and is now undergoing Phase 3 trials in sub-Saharan Africa. Meanwhile, a single gene therapy injection given at birth showed promise in protecting primates from infection for at least three years in preclinical research. If this solution advances, it could be a gamechanger for preventing mother-to-child HIV transmission.Broadly neutralizing antibodies for long-term HIV control and cureBroadly neutralizing antibodies (bnAbs), which are made naturally by some individuals and target parts of the HIV virus that are less prone to mutation, are also showing promise in HIV treatment, prevention, and cure strategies. Studies have shown that bnAbs can effectively neutralize a broad range of HIV strains and bnAb combinations can maintain viral suppression in the absence of antiretroviral therapy, but challenges remain around their cost, global scalability, and the potential for viral resistance.Researchers are testing combination approaches with other drugs to potentially enhance the efficacy of bnAbs in controlling HIV. Study results presented earlier this year found that more than half of study participants living with HIV who received a pair of bnAbs plus the drug N-803 experienced delayed viral rebound or maintained a low or suppressed viral load during interruption from antiretroviral treatment. N-803, which is approved as a treatment for bladder cancer, appears to help reactivate and eliminate dormant HIV virus.Gilead also shared promising results from a Phase 2 trial earlier this year combining lenacapavir with two bnAbs as treatment for HIV. This regimen is the most advanced bnAbs-based treatment to date, with Phase 3 trials expected to start soon.Beyond treatment, many leading HIV scientists believe that a vaccine that elicits the production of bnAbs could be key to targeting the tricky, mutating virus. IAVI and partners developed immunogens, a type of antigen that can trigger an immune response, for use in a vaccine strategy to induce bnAb production, which demonstrated success in several Phase 1 clinical trials. A new study looking at data from two of the trials shows that this targeted vaccine strategy can successfully activate early immune responses relevant to HIV, an important step toward an HIV vaccine. The candidates use an mRNA vaccine platform, enabling faster production and clinical testing.While bnAbs have the potential to transform HIV prevention and treatment, further research is needed to overcome cost and delivery barriers to ensure that future innovations can be rapidly scaled up and distributed to the communities who can benefit most from them.While global HIV efforts have faced setbacks this year, we’ve also seen the extraordinary progress that’s possible when partners unite around innovation. New tools and approaches are emerging that could change the course of the global HIV/AIDS pandemic, preventing millions of new infections and deaths every year. But their impact depends on what happens next. To realize their full potential, countries, donors, and partners must come together and recommit to fund research, prioritize equitable access, and accelerate the scale-up of new tools. The future of the HIV/AIDS response hinges on how we meet this moment.

This month marks 35 years since the World Summit for Children, the first time in history when a meeting of heads of state and government was held exclusively to address the well-being of children. At this landmark event in New York City, world leaders adopted a Declaration on the Survival, Protection, and Development of Children and a Plan of Action for its implementation, which made specific recommendations in a variety of areas, including child and maternal health and family planning. We’ve made significant progress in children’s health globally since then—the total number of deaths of children under five worldwide has declined from 12.8 million in 1990 to 4.8 million in 2023. The number of neonatal deaths (deaths in the first 28 days) has also dropped, although more slowly, from 5.2 million in 1990 to 2.3 million in 2023. However, there are still major challenges in children’s health, especially in the Africa region, where children continue to have the highest risk of dying before reaching age five. Infectious diseases, including acute respiratory infections, diarrhea, and malaria, as well as birth complications, remain among the leading causes of death for children under five. More than half of child deaths are due to conditions that could be prevented and treated with adequate access to health care or other quality of life improvements, particularly in that crucial first month: neonatal deaths make up 47 percent of under-five child deaths.Another challenge is the lack of medicines designed specifically for children—only 30 percent of essential medicines are available in child-appropriate formulations. Instead, children are often treated with imprecise partial doses of medicines that were designed and tested in adults, which can lead to toxicity and complications. This gap results from a variety of barriers: it can take up to 25 years to bring a pediatric medicine to market and only one in ten clinical trials registered between 2000 and 2023 included children. Innovative technologies rolled out since 1990 have already saved millions of lives, and new tools in the pipeline designed with the needs of children, and particularly the most vulnerable kids, in mind could bring down child mortality even more. Diarrheal disease vaccines While diarrheal-disease related deaths among children under five have decreased drastically since 1990, down an estimated 79 percent from 1990 to 2021, they still cause a severe burden; 4.8 million children under five died in 2023. Rotavirus, Shigella, and enterotoxigenic Escherichia coli (ETEC) are among the common pathogens that can lead to diarrheal disease.  The first rotavirus vaccines of the generation still in use today become available just under 20 years ago, approved in the United States in 2006 and later recommended by the World Health Organization for all children. Today, these vaccines are used in more than 110 countries around the world and have contributed to a significant drop in child hospitalizations and deaths from diarrhea. Rotavirus vaccines have had a marked impact on child mortality, preventing 139,000 deaths in children under five between 2006 and 2019. However, rotaviruses are still the leading cause of severe diarrhea in children under five in low- and middle-income countries, pointing to the need for increased access to existing vaccines, as well as new and more effective ones.Unfortunately, approved vaccines don’t yet exist for ETEC and Shigella, though promising candidates are in development. PATH is working with partners to advance vaccines against these bacteria, which could help save thousands of additional lives. For Shigella, PATH is working with the US Naval Medical Research Command and the Walter Reed Army Institute of Research to advance a vaccine candidate, which demonstrated strong safety and efficacy in a recent Phase 1 trial. PATH is also working with these US partners, alongside Sanofi Pasteur and the Infectious Disease Research Institute, to advance an ETEC vaccine candidate, which displayed strong potential in a Phase 1 trial in adults, paving the way for the development of a vaccine for infants and young children, who face the highest risk. If they continue to prove successful, these vaccines could have a tremendous public health impact, not only preventing deaths but also reducing the long-term health and economic effects of malnutrition and developmental delays associated with experiencing severe diarrhea, as well as limiting the growth of antimicrobial resistance.  Pediatric products for malaria and more Children under five account for nearly 80 percent of global malaria deaths. Despite this significant burden, for a long time, there was a lack of a suitable malaria medicines for children, who were treated with crushed tablets designed for adults. The first child-friendly, quality-approved malaria treatment, launched in 2009 by Medicines for Malaria Venture (MMV) and Novartis, is sweet-tasting and quickly dissolves in water. 500 million courses of Coartem® Dispersible have been delivered worldwide as of this April, saving more than 1.8 million lives. MMV also delivered the first malaria treatment for newborn babies and young infants, which is an adapted version of Coartem® that was approved earlier this year. The two first-ever malaria vaccines, approved in the last five years, have been another major game changer for children with malaria, with 21 countries in Africa having introduced them so far, alongside other complementary prevention methods like chemoprevention and vector controlMMV is also working with partner Eyam Health to develop next-generation monoclonal antibody-based approaches to create affordable, long-acting malaria preventative therapies and treatments. Eyam’s technology has the potential to deliver future durable therapies without the need for cold storage and at an extremely low cost, projecting that a single long-acting therapeutic injection could provide protection for more than 50 million children and pregnant women for the months-long rainy season, which is peak time for malaria transmission. This would significantly simplify seasonal malaria chemoprevention, which today involves taking a course of oral drugs monthly for up to five months. Alongside these innovations for malaria, child-friendly medicines for HIV and neglected tropical diseases are also helping address the gap of suitable medicines for children and reduce the burden of treatment for children and their caregivers.  Diagnostics for pre- and postnatal complications and infections Premature birth, birth complications, and neonatal infections are among the leading causes of neonatal deaths, often resulting from limited access to timely screening, particularly in low-resource settings that face a lack of health care services, poor laboratory infrastructure and insufficiently skilled health care workers. Low-cost, easy-to-use diagnostics that can be deployed at the point of care are helping ensure that mothers and babies everywhere have access to routine screening in the pre- and postnatal periods, helping catch and address complications and infections earlier. One innovation is a portable, user-friendly ultrasound developed by Philips that connects to a mobile app on a phone or tablet, helping bring high-quality diagnostics to the point of care. The technology is being rolled out in low- and middle-income countries with the support of the Gates Foundation and has already demonstrated its ability to improve informed decision-making when triaging pregnant women in rural underserved communities.Bacterial infections caused by pneumonia, sepsis, and meningitis are the primary cause of neonatal infections, and most could be averted with adequate access to preventive interventions, early diagnosis, and care. Almost half of sepsis cases occur in children under five. The Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator has awarded funding to Melio, AstraDx, and QuantaMatrix to demonstrate feasibility of their rapid diagnostics for neonatal sepsis, all of which aim to provide results in mere hours, down from days. Early diagnosis is particularly important because the risk of death from neonatal sepsis increases about 7.6 percent every hour treatment is delayed. These innovations have the potential to help greatly reduce the risk of babies and mothers dying from complications and infections during the critical periods before and after birth.  Thirty-five years since the World Summit for Children, we’ve made tremendous progress in children’s health. Investing in innovative technologies specifically designed for children and striving to ensure children and their needs are considered and included in research across health areas will set up future generations for an even healthier future.