U.S. FDA Accepts Gilead’s New Drug Applications for Twice-Yearly Lenacapavir for HIV Prevention Under Priority Review

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) submissions for lenacapavir—the company’s twice-yearly injectable HIV-1 capsid inhibitor—for the prevention of HIV as pre-exposure prophylaxis (PrEP).

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.

“Today, 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 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.

A bout Lenacapavir

Lenacapavir is approved in multiple countries for the treatment of adults with multi-drug resistant HIV in combination with other antiretrovirals. The use of lenacapavir for HIV prevention is investigational and the safety and efficacy of lenacapavir for this use have not been established.

The multi-stage mechanism of action of lenacapavir is distinguishable from other currently approved classes of antiviral agents. While most antivirals act on just one stage of viral replication, lenacapavir is designed to inhibit HIV at multiple stages of its lifecycle and has no known cross resistance exhibited in vitro to other existing drug classes.

Lenacapavir is being evaluated as a long-acting option in multiple ongoing and planned early and late-stage clinical studies in Gilead’s HIV prevention and treatment research program. Lenacapavir is being developed as a foundation for potential future HIV therapies with the goal of offering both long-acting oral and injectable options with several dosing frequencies, in combination or as a mono agent, that help address individual needs and preferences of people and communities affected by HIV.

About the PURPOSE Program

Gilead’s landmark PURPOSE program is the most comprehensive and diverse HIV prevention trial program ever conducted. The program comprises five HIV prevention trials around the world that are focused on innovation in science, trial design, community engagement and health equity.

The PURPOSE trials are evaluating the safety and efficacy of an investigational, twice-yearly injectable medicine, lenacapavir, to reduce the chance of getting HIV. The Phase 2 and 3 program, consisting of PURPOSE 1-5, is assessing the potential of lenacapavir to help a diverse range of people around the world who could benefit from PrEP.

More information about the PURPOSE program, including individual trial descriptions, populations and locations, can be found at www.purposestudies.com.

About Gilead HIV

For more than 35 years, Gilead has been a leading innovator in the field of HIV, driving advances in treatment, prevention and cure research. Gilead researchers have developed 12 HIV medications, including the first single-tablet regimen to treat HIV, the first antiretroviral for pre-exposure prophylaxis (PrEP) to help reduce new HIV infections, and the first long-acting injectable HIV treatment medication administered twice-yearly. Our advances in medical research have helped to transform HIV into a treatable, preventable, chronic condition for millions of people.

Gilead is committed to continued scientific innovation to provide solutions for the evolving needs of people affected by HIV around the world. Through partnerships, collaborations and charitable giving, the company also aims to improve education, expand access and address barriers to care, with the goal of ending the HIV epidemic for everyone, everywhere. Gilead was recognized as one of the leading philanthropic funders of HIV-related programs in a report released by Funders Concerned About AIDS.

Forward-Looking Statements

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 that are subject to risks, uncertainties and other factors, including Gilead’s ability to initiate, progress and complete clinical trials in the anticipated timelines or at all, and the possibility of unfavorable results from ongoing and additional clinical trials, including those involving lenacapavir (such as PURPOSE 1 and PURPOSE 2); uncertainties relating to regulatory applications and related filing and approval timelines, including the New Drug Applications seeking FDA approval and the MAA and EU-M4all seeking EMA approval of lenacapavir for PrEP, and the risk that any regulatory approvals, if granted, may be subject to significant limitations on use or subject to withdrawal or other adverse actions by the applicable regulatory authority; the possibility that Gilead may make a strategic decision to discontinue development of lenacapavir for indications currently under evaluation and, as a result, lenacapavir may never be successfully commercialized for such indications; Gilead’s ability to effectively manage the access strategy relating to lenacapavir, subject to necessary regulatory approvals; and any assumptions underlying any of the foregoing. These and other risks, uncertainties and factors are described in detail in Gilead’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, as filed with the U.S. Securities and Exchange Commission. These risks, uncertainties and other factors could cause actual results to differ materially from those referred to in the forward-looking statements. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The reader is cautioned that any such forward-looking statements are not guarantees of future performance and involve risks and uncertainties, and is cautioned not to place undue reliance on these forward-looking statements. All forward-looking statements are based on information currently available to Gilead, and Gilead assumes no obligation and disclaims any intent to update any such forward-looking statements.

U.S. full Prescribing Information for Truvada, including Boxed Warning, and lenacapavir are available at www.gilead.com .

Gilead and the Gilead logo, Truvada, and Truvada for PrEP are registered trademarks of Gilead Sciences, Inc., or its related companies.

For more information about Gilead, please visit the company’s website at www.gilead.com , follow Gilead on X/Twitter (@Gilead Sciences) and LinkedIn (@Gilead-Sciences).