Breakthroughs Blog: FDA

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. 

Interested in more global health innovation news? Every week GHTC scours media reports worldwide to deliver essential global health R&D news and content to your inbox. Sign up now to receive our weekly R&D News Roundup email. Last Wednesday, Gilead announced that its twice-yearly injectable drug for HIV prevention has received approval from the US Food and Drug Administration, the first and only such shot for HIV prevention. The drug, lenacapavir, which is already used to treat HIV in certain populations, was found to dramatically reduce the risk of infection, providing near total protection among cisgender men and women, transgender men and women, and nonbinary people in clinical trials. The twice-a-year injection offers a significant improvement over current HIV prevention options that require a daily pill or every two-month injections, helping improve uptake and adherence by providing a more discreet, private, and less cumbersome alternative. Pricing and access plans for the United States and the rest of the world will determine the scale of lenacapavir’s impact on the broader HIV epidemic.Researchers from Cornell University have found that the antibiotic rifampin, which is already widely used, is 99.9 percent effective against Salmonella Typhi, the bacterium that causes typhoid fever, which a growing threat due to antibiotic resistance. The researchers found that S. Typhi is still highly susceptible to the antibiotic and that rifampin-resistant typhoid is extremely unlikely to emerge, despite concern due to the previous emergence of rifampin-resistant tuberculosis cases. The researchers found that rifampin may also be effective against other life-threatening diseases like bacterial pneumonia and meningitis.Newly published research found that a simple, affordable device made with strips of paper was more effective than other commonly used testing methods at detecting malaria in asymptomatic people in Ghana. Malaria testing, particularly at the point of care, is essential to malaria control in endemic areas, especially as increased uptake of the new preventive malaria vaccine lowers natural immunity, raising the need for widespread surveillance. The new test is highly sensitive, offers results in about 30 minutes, and can detect even a low density of parasites in the blood, catching malaria before a patient is sick.

The recent staffing cuts at the U.S. Food and Drug Administration (FDA), which affected roughly 20 percent of its workforce, have prompted concerns about the agency’s ability to fulfill its mission. The cuts would severely impact US companies and US global leadership in the biomedical space.  The global implications would be even more grievous—particularly for low- and middle-income countries (LMICs) that depend on the FDA’s regulatory leadership. Many LMICs and other countries rely on the FDA’s assessments, approvals, and post-market surveillance data to inform their own regulatory decisions. These programs, often referred to as “reliance programs,” streamline national approval processes by referencing trusted decisions from agencies like the FDA. As the agency grapples with significant staffing reductions, there could be ripple effects on these international structures.What are reliance programs?Reliance programs allow regulatory authorities in one country to draw on the evaluations, inspections, and approvals of trusted agencies—known as Stringent Regulatory Authorities (SRAs)—such as the FDA. Many LMICs use FDA decisions to streamline access to essential medicines and vaccines, avoiding duplicative reviews and accelerating timelines. This model is especially valuable where regulatory resources are limited, allowing local agencies to focus on post-market surveillance and health care delivery. The FDA’s assessments serve as a de facto quality mark, reinforcing confidence in products worldwide.One example is the World Health Organization’s (WHO’s) Collaborative Registration Procedure (CRP), which enables countries to fast-track the registration of WHO-prequalified medicines by leveraging FDA and other SRA reviews. Between 2015 and 2021, the CRP facilitated 59 approvals for 16 medicines across 23 countries, demonstrating its impact on timely access to quality-assured medicines for patients.Implications for the United StatesIf the FDA’s reduced capacity compromises its ability to effectively conduct timely reviews, update guidance documents, or perform inspections, the most immediate impacts will be felt in the United States. The United States could lose influence in setting international safety, efficacy, and quality standards if other regulatory authorities begin supplanting the FDA's role in global reliance networks. Over time, this erosion of regulatory leadership could diminish the FDA’s status as the gold standard globally—a position that has long bolstered American soft power in global health and trade. Manufacturers based in the United States that are developing products for diseases endemic in LMICs may also find their innovations at a disadvantage if the FDA lags behind other regulators in review speed or capacity or if the WHO prequalification program, which allows US manufacturers to leverage mechanisms that streamline and accelerate the deployment of their innovations in LMICs,  no longer recognizes the validity of FDA review. In 2024, for example, WHO prequalified a Hepatitis C self-test manufactured by Bethlehem, Pennsylvania-based OraSure Technologies. An innovative mpox point-of-care molecular test developed by Sunnyvale, California-based Cepheid became the first such diagnostic ever approved by WHO for emergency use. These FDA-WHO collaborations are vital for the purchase and procurement of American products. If the FDA retreats from this space, it could reduce the competitiveness of American firms in emerging markets and make the United States a less attractive regulatory partner for international collaboration. Preserving the FDA’s global standing is, therefore, not only a matter of public health but also of economic and geopolitical significance.Potential impacts on LMICsHowever, the secondary consequences could be more acute for countries relying on the FDA’s efficiency to inform their regulatory decisions. Delays in product approvals may extend the time before essential medicines reach patients in LMICs—where the burden of disease is often highest and timely access is critical. The loss of experienced personnel and institutional knowledge may also weaken confidence in the consistency and rigor of FDA assessments, prompting some regulators to reevaluate the extent of their reliance. The FDA should build its internal capacity to address neglected diseases rather than reduce staff, providing training opportunities and ensuring their workforce has the prerequisite expertise.Looking aheadTo help safeguard both domestic and global health outcomes, it will be important for the FDA to maintain its capacity to carry out timely, science-based evaluations. One upcoming opportunity to support this effort is the Prescription Drug User Fee Act (PDUFA) reauthorization, which allows the FDA to collect fees from pharmaceutical companies to help fund its drug review programs. PDUFA has long been a bipartisan priority and is key in supporting the agency’s scientific workforce and review capabilities. Ensuring the continuity of this program will help the FDA meet its responsibilities at home and abroad. As global reliance on the FDA remains high—particularly among countries with limited regulatory resources—continued investment in the agency’s capacity will be necessary both for US innovation and global health systems that depend on its leadership.Now more than ever, we must protect the FDA’s ability to lead. A weakened FDA puts American innovation and jobs at risk, slows access to breakthrough products, and opens the door for other countries to set the standards we once defined. The world looks to the FDA, not just for safety and science, but for leadership. With PDUFA reauthorization on the horizon, Congress has a critical opportunity to invest in the FDA’s workforce and secure the United States’ role as the global leader in health and biomedical innovation.

Interested in more global health innovation news? Every week GHTC scours media reports worldwide to deliver essential global health R&D news and content to your inbox. Sign up now to receive our weekly R&D News Roundup email. A new study led by a multi-country research team has found that the Xpert MTB/Ultra molecular diagnostic test for stool samples, which is currently only recommended for tuberculosis (TB) diagnosis in children, could serve as an alternative TB diagnosis option for adults living with HIV—a potential gamechanger for improving diagnosis among this population. Thirteen percent of the 1.25 million TB deaths in 2012 were among people living with HIV, but access to the sputum- and urine-based tests currently recommended for TB diagnosis in this population is limited in many settings, and they are not effective for all people living with HIV. The researchers found that, compared to current tests, the stool test was effective, and, in some cases, even identified additional cases that were not detected in sputum or bacterial culture.The Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator (CARB-X) last week announced a $3.7 million award to ArrePath to support its first-in-class antibiotic for treating complicated urinary tract infections caused by multidrug-resistant Enterobacterales bacteria. If it proves successful, the novel antibiotic class could target the vast majority of multidrug-resistant Enterobacterales infections. The class of antibiotics also has attributes that suggest it could be administered orally or intravenously, allowing more flexibility for patients. ArrePath identified the new class using an artificial intelligence and machine-learning-enabled approach.Last week, the US Food and Drug Administration approved a freeze-dried formulation of Bavarian Nordic’s vaccine for mpox and smallpox, Jynneos, enabling additional flexibility in stockpiling doses of the vaccine to prepare for future outbreaks of smallpox or mpox. The freeze-dried vaccines are easier to store and transport and have a longer shelf life than the frozen liquid version, which was approved in 2019. Bavarian Nordic will deliver the first freeze-dried vaccines later this year.