Breakthroughs Blog: WHO

Recently published results from a Phase 2b trial testing a new regimen for people with chronic Trypanosoma cruzi infection could offer a safer, more practical treatment option for the infection, which affects more than seven million people globally, mainly in Latin America, and can develop into Chagas disease. Compared to the current standard regimen that involves twice-a-day treatment for 60 days and is frequently accompanied by uncomfortable side effects, leading to treatment failure, the new regimen involves a 30-day course taken once daily. Trial participants treated with the shorter regimen had significantly reduced side effects, found it easier to follow, and saw lower treatment interruption. If it continues to demonstrate success in large Phase 3 trials, the regimen could transform treatment for patients globally, as T. cruzi infection becomes a growing challenge in the United States, Europe, and Japan.The first participants have been vaccinated in a trial in the Democratic Republic of the Congo (DRC), where the country’s ongoing outbreak of the Bundibugyo strain of Ebola has now become the largest Ebola outbreak in the country’s history and the second largest in history. Health workers in Bunia, a DRC city at the center of the outbreak, were the first to be vaccinated in the real-world efficacy trial evaluating whether the Ervebo vaccine, which is designed to target the more common Zaire strain, offers some cross-protection against Bundibugyo. There are no approved vaccines for Bundibugyo Ebola, and there were no candidates in late-stage development before the start of the outbreak of the rarer strain of the virus, which has only caused three outbreaks since it was first discovered around 20 years ago. Since the outbreak began, global partners have fast-tracked the development of earlier-stage candidates, but researchers hope the Ervebo vaccine could provide some protection before those candidates are ready for evaluation in DRC. As artificial intelligence (AI) continues to rapidly transform many aspects of technology and science, including health research, a new report from the World Health Organization (WHO) speaks to how stakeholders can help ensure AI-enabled health research is conducted responsibly and benefits everyone. According to the report, while AI has the potential to accelerate health innovation and improve health outcomes, it also presents risks related to equity, privacy, safety, transparency, and more. WHO suggests that researchers, ethics committees, regulators, funders, and policymakers can act to strengthen ethical oversight of AI throughout the health research continuum. WHO also notes that most AI development remains concentrated in high-income settings and contends that strengthening leadership, capacity, and participation in AI in low- and middle-income countries will be important to ensuring the technology does not exacerbate existing inequities in health and health research. 

Last week, Gilead Sciences announced that it has expanded the scope of its voluntary licensing agreements with six manufacturing partners, who are already manufacturing the recently approved twice-yearly lenacapavir for HIV prevention, to include investigational once-yearly lenacapavir. Following the unprecedented Phase 3 trials for twice-yearly lenacapavir, an ongoing Phase 3 trial is evaluating a once-yearly option that could provide a full year of prevention with just one annual dose, further expanding prevention options and addressing persistent barriers to uptake and adherence. Establishing these agreements at this early stage helps manufacturers, regulators, health systems, and communities prepare for the broad, rapid, and equitable scale-up of the once-yearly option, if it is eventually approved. The World Health Organization (WHO) has updated its malaria guidelines to include Coartem® Baby, the first antimalarial developed specifically for newborns and young infants weighing between two and five kilograms. This patient group previously had no medicine specifically designed for their unique needs, leaving them to be treated with modified doses of formulations intended for older children, raising the risk of treatment failure or toxicity. The drug was developed by Novartis and partner Medicines for Malaria Venture (MMV), with financial support from the European & Developing Countries Clinical Trials Partnership and the Swedish International Development Cooperation Agency. WHO’s inclusion of Coartem® Baby in the guidelines informs and enables malaria-endemic countries to introduce the treatment in national malaria programs, supporting broader access. After receiving prequalification by WHO and approval from Swiss and Ghanian regulatory authorities, Coartem® Baby has so far been rolled out in 11 countries. The Coalition for Epidemic Preparedness Innovations (CEPI) recently announced that it is awarding $3.9 million to support research projects aimed at improving understanding of the Bundibugyo ebolavirus behind the ongoing outbreak in the Democratic Republic of the Congo. Specifically, the funding will support modelling and analytic teams led by the Institut National de Recherche Biomédicale, Imperial College London, and Makerere University School of Public Health. These teams will collect, integrate, and analyze outbreak data to guide the outbreak response; inform the design of fast, efficient vaccine clinical trials; and support longer-term preparedness against similar outbreaks in the region. To ensure long-term applicability, the partners have placed a strong emphasis on capacity strengthening and open sharing of data and methods, so that the tools and knowledge these projects generate can be utilized in future outbreaks in the region. 

Fungi have existed for hundreds of millions of years, long before dinosaurs walked the Earth, but fungal infections have become a rapidly growing challenge for human health in recent decades.Fungal diseases affect more than 300 million people each year, disproportionately impacting people in low-resource settings and those with weakened immune systems, including people living with HIV, cancer patients, and transplant recipients. Most fungal infections affect superficial tissue like the skin, nails, or hair and can cause chronic illness and productivity loss. Though less common, invasive fungal infections can spread throughout the body and are associated with high mortality.Why fungal diseases are becoming harder to ignoreThe rising incidence of fungal infections is driven by a variety of factors, including conflict, poverty, urbanization, pollution, travel, and climate change, which increase exposure, heighten vulnerability, and expand the reach of dangerous fungal pathogens. At the same time, advances in medicine mean more people are living longer with conditions that weaken the immune system, increasing the number of people at greater risk. Growing antifungal resistance is also making infections harder to treat and further limiting already scarce treatment options.Despite the mounting burden, fungal disease remains underprioritized, underdiagnosed, under-surveilled, and underfunded, making it one of the world’s most neglected global health threats.Why innovation is falling behindFungal infections receive less than 1.5 percent of all infectious disease research and development (R&D) funding, contributing to sparse pipelines for both therapeutics and diagnostics. Closing these gaps will require greater investment in R&D, particularly for tools designed for resource-constrained settings and targeting the pathogens that pose the greatest threat to human health. Stronger regulatory and procurement systems and international collaboration will also be essential to ensure emerging tools are affordable and accessible worldwide.Despite persistent gaps, several promising innovations demonstrate the potential for R&D to transform the global response to fungal disease.Promising advances in antifungal treatmentThere are currently only four classes of antifungal medicine available, many of which are associated with serious side effects or require lengthy, hospital-administered treatment courses. The pipeline of new antifungal therapies also remains thin.The World Health Organization (WHO) reported last year that only nine antifungal therapies were in clinical development to address WHO’s designated top four priority pathogens, including three in Phase 3 development. Although the Phase 3 trial for one of those candidates has since been terminated, another candidate, olorofim, developed by Shionogi and F2G, recently reported positive Phase 3 trial results. If approved, it could become the first antifungal treatment with a novel mechanism of action for that infection in more than 20 years.Earlier-stage research is also yielding promising advances. Researchers recently discovered that an antibiotic could target a priority fungal pathogen by switching it back from its invasive form to a less harmful form. This antibiotic could help existing antifungal drugs target dangerous fungal pathogens, allowing lower, less toxic doses of those drugs to be used.Together, these advances offer hope of a new generation of antifungal therapies.The future of fungal diagnosticsDiagnostic tools for critical fungal pathogens exist but with significant limitations, including invasive sampling requirements, long turnaround times, limited pathogen coverage, and inadequate sensitivity. Many also depend on sophisticated laboratories and highly trained staff, making them difficult to deploy in resource-constrained settings globally that bear a disproportionate burden of fungal disease.The MycoMEIA® assay developed at Johns Hopkins University and commercialized by Pearl Diagnostics represents a recent advance in antifungal diagnostics. The test received US Food and Drug Administration clearance in 2025. It uses a urine sample, which is much simpler and less invasive for patients than current options, and provides results in just a few hours.Another promising research development illustrates where fungal diagnostics may be headed next. Researchers at the Mayo Clinic and Zepto Life Technology recently reported early results from a new diagnostic platform that was able to identify 18 different fungal pathogens with a single test. While still in development, such approaches could not only streamline diagnosis for health care providers and patients, but also reduce costs and resource requirements, making them well-suited for resource-constrained settings.Closing the innovation gap Fungal diseases and antifungal resistance are attracting greater international attention, but the response still lags behind the scale and pace of this growing threat. Increased investment in the R&D of fit-for-purpose innovations, paired with stronger regulatory and health systems and global coordination, will be crucial to ensure new diagnostics and treatments emerge from the pipeline and reach the patients who need them most. 

Few diseases have shaped global health like the HIV/AIDS pandemic. Since it began, more than 40 million lives have been lost, leaving an enduring impact on families, communities, and health systems worldwide. But HIV has also become one of the clearest examples of what sustained research and development (R&D) can achieve. From antiretroviral therapies (ART) to long-acting prevention tools, decades of scientific innovation have fundamentally changed what is possible in the fight against HIV.This year, HIV innovation stands at a pivotal moment. Shifting funding landscapes and evolving global health priorities have raised important questions about how R&D for HIV will be sustained in the years ahead. As global leaders have reaffirmed the importance of continued R&D investment—such as through the recently adopted United Nations High Level Meeting political declaration on HIV/AIDS—we must also focus on the systems and programs that translate scientific breakthroughs into impact. Few advances better illustrate this balance than lenacapavir, or LEN, an injectable form of HIV prevention requiring only two injections per year, spaced 6 months apart. Unlike daily oral pre-exposure prophylaxis (PrEP), which depends on consistent adherence, LEN offers protection by eliminating daily pill burden for consumers and communities vulnerable to HIV.The journey of LEN reflects the long arc of HIV innovation. Decades of research supported by the US National Institutes of Health helped establish the HIV capsid as a promising drug target, creating the foundation for a new class of HIV medicines. Then, in 2006, researchers at Gilead Sciences began developing capsid inhibitors designed to suppress HIV replication while offering the potential for long-acting treatment. At the time, ART had transformed HIV from a fatal disease into a manageable chronic condition for millions of people, but treatment relied on daily medication, creating ongoing challenges for adherence and access.Following years of research and clinical development, the US Food and Drug Administration (FDA) approved LEN in 2022 as a treatment for adults living with multidrug-resistant HIV whose existing treatment regimens were no longer effective. While this approval marked an important milestone, researchers at Gilead continued exploring the drug's potential as a long-acting option for HIV prevention and found remarkable resultsThe first pivotal late-stage prevention trial of the drug, called PURPOSE 1, conducted among women in South Africa and Uganda—including pregnant and lactating women—found that LEN provided near-complete protection against HIV infection and outperformed daily oral PrEP. PURPOSE 2, a subsequent study, conducted across seven countries and including men and gender-diverse people, confirmed similarly high levels of protection in late 2024. Beyond their clinical success, these studies also represented an important step toward more inclusive HIV research by intentionally enrolling populations that have been historically underrepresented in clinical trials.  Just six months after Gilead submitted LEN for review as an HIV prevention tool, the FDA approved the drug in June 2025 under a priority review designation pathway. Because regulators were able to rely on the extensive safety data generated during the drug's earlier approval for HIV treatment, they could focus their review on its use as a novel preventive intervention. The result was an efficient regulatory process that maintained rigorous standards while accelerating future access to an important new prevention tool. Swift regulatory action extended beyond the United States. In October 2025, the World Health Organization (WHO) prequalified LEN in just 36 days using its abridged prequalification pathway. This expedited process enabled WHO to leverage assessments already completed by the European Medicines Agency’s (EMA) European Union-Medicines for all program, which provides scientific opinions on medicines for use in low- and middle-income countries, while conducting its own review focused on issues relevant to the global procurement and implementation of LEN.  This rapid review mattered because WHO prequalification serves as a critical gateway for procurement by many international organizations and countries, particularly in low- and middle-income countries. Accelerating this process helps ensure that countries are not waiting months or years for access after a product has already demonstrated its safety and effectiveness through other rigorous stringent regulatory approval processes.Accelerated regulatory approval and collaboration continued across multiple regions. Following FDA, EMA, and WHO approval, LEN rapidly advanced through regulatory processes in South Africa, Australia, Brazil, and Canada, and, as of today, it is approved by 17 regulatory bodies, including 12 countries in Africa. In several cases, regulators leveraged reliance mechanisms, such as WHO’s collaborative registration procedure, international collaboration, and priority review pathways to streamline review and accelerate access to this important breakthrough without compromising regulatory rigor. Swift regulatory action was soon matched by commitments from the United States; the Global Fund to Fight AIDS, Tuberculosis and Malaria; and Gilead to rapidly scale up access to LEN and accelerate generic production in high-burden countries.This coordinated approach, supported by political commitment and strategic public-private partnership, created an unusually efficient pathway from scientific discovery to implementation. Compared with earlier HIV prevention technologies, such as oral PrEP and the dapivirine vaginal ring, LEN has progressed through key milestones at a rapid pace. This allowed for accelerated product introduction of lifesaving technologies at the country level, while simultaneously shaping the market and demand for LEN and future prevention options. While every health technology faces unique scientific, regulatory, and access considerations, LEN demonstrates how thoughtful planning, regulatory preparedness, and collaboration between researchers, regulators, countries, multilateral organizations, and communities can significantly shorten the time between discovery and access.As conversations about the future of HIV innovation continue, LEN offers an important blueprint for global health innovation more broadly. It reminds us that sustained investment in R&D produces transformative breakthroughs, inclusive clinical trials generate stronger science, and innovative regulatory pathways can accelerate, rather than compromise, rigorous review. Moreover, the accelerated path from research to rollout illustrates the coordination and collaboration required to advance technologies through regulatory approval while simultaneously shaping markets to enable introduction at scale. The lessons from LEN are already informing the next generation of HIV prevention innovations, with developers pursuing earlier access and licensing strategies for products such as Merck's MK-8527. While the US government, the Global Fund, and Gilead have recently expanded commitments to scale up LEN, the ultimate measure of success will be whether LEN—and the innovations that follow it—reach the people who need them most.  The future of innovations for HIV, as well as tuberculosis, malaria, emerging threats with pandemic potential, and beyond, will require the same integrated approach. Scientific discovery, product development, efficient regulatory pathways, and equitable access cannot be treated as separate objectives; they are mutually reinforcing components. Innovation succeeds not when a product is approved, but when it reaches the communities whose lives it has the power to change. LEN has shown us what’s possible when the entire R&D ecosystem works together. The challenge now is ensuring that this success becomes the standard rather than the exception.