Gilead new drug applications for twice-yearly Lenacapavir for HIV Prevention accepted by USFDA under priority review

The FDA will review the applications under priority review and has assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 19, 2025. Acceptance of the NDAs follows receipt of Breakthrough Therapy Designation for lenacapavir for PrEP granted by the FDA in October 2024. The Breakthrough Therapy Designation process is intended to expedite the development and regulatory review of investigational treatments for serious or life-threatening conditions that, based on preliminary clinical evidence, have the potential to substantially improve clinical outcomes compared with available therapy.

“We are one step closer to introducing the first-ever twice-yearly HIV prevention choice that could, if approved, help transform the landscape for individuals who need or want additional prevention options that better fit into their lives,” said Dietmar Berger, MD, PhD, Chief Medical Officer, Gilead Sciences. “We’re excited about the potential of lenacapavir to make a real difference in HIV prevention in the U.S. and around the world, supporting the broader goal of ending the HIV epidemic for everyone, everywhere.”

The NDAs are based on data from the Phase 3 PURPOSE 1 and PURPOSE 2 trials conducted by Gilead. In PURPOSE 1 (NCT04994509), data showed twice-yearly lenacapavir demonstrated zero infections in the lenacapavir group and 100% risk reduction and superiority compared to background HIV incidence (bHIV) for the investigational use of HIV prevention in cisgender women. In PURPOSE 2 (NCT04925752), there were two HIV infections in the lenacapavir group, demonstrating 99.9% of participants in the lenacapavir group did not acquire HIV infection, a 96% risk reduction in HIV infections, and superiority compared to bHIV among a broad and geographically diverse range of cisgender men and gender-diverse people. In both trials, twice-yearly lenacapavir also demonstrated superiority of prevention of HIV infections when compared with once-daily oral Truvada (emtricitabine 200mg and tenofovir disoproxil fumarate 300mg; F/TDF) and was generally well-tolerated, with no significant or new safety concerns identified. Based in part on these trial results, Science Magazine in December 2024 named lenacapavir its 2024 “Breakthrough of the Year.”

"Gilead is executing an access strategy, informed by global health advocates and organizations, that prioritizes speed and enables the most efficient paths for the regulatory review, approval of and access to lenacapavir for PrEP in regions around the world. As part of that strategy, earlier this year Gilead submitted a European Commission marketing authorization application (MAA) and an EU-Medicines for All (EU-M4all) application to the European Medicines Agency (EMA) for lenacapavir for PrEP. The EU-M4all application aims to help facilitate availability of lenacapavir for PrEP in low- and lower-middle-income countries. Through the EU-M4all procedure, national regulatory authorities in these regions can leverage the EU opinion to expedite their review processes, potentially accelerating access to lenacapavir for PrEP. Updates on regulatory filings for lenacapavir for PrEP will be shared as discussions with regulatory bodies progress," the Company stated in a release.

The use of lenacapavir for the prevention of HIV is investigational and is not approved anywhere globally. The safety and efficacy of this use has not been established by the U.S. FDA.

There is currently no cure for HIV or AIDS.