U.S. FDA Accepts Gilead’s New Drug Applications for Twice-Yearly Lenacapavir for HIV Prevention Under Priority Review
Gilead Sciences announced that the FDA has accepted its New Drug Application for lenacapavir, a twice-yearly injectable HIV prevention treatment, under priority review with a PDUFA date of June 19, 2025. The application follows Breakthrough Therapy Designation granted in October 2024.
The NDAs are supported by Phase 3 trials PURPOSE 1 and PURPOSE 2. PURPOSE 1 showed 100% risk reduction in cisgender women, with zero infections in the lenacapavir group. PURPOSE 2 demonstrated 99.9% effectiveness with a 96% risk reduction among cisgender men and gender-diverse people, with only two HIV infections reported.
Gilead has also submitted applications to the European Medicines Agency for review under accelerated assessment, including an EU-M4all application to facilitate availability in low and lower-middle-income countries.
Gilead Sciences ha annunciato che la FDA ha accettato la sua Richiesta di Nuovo Farmaco per lenacapavir, un trattamento iniettabile per la prevenzione dell'HIV da somministrare due volte l'anno, sotto revisione prioritaria con una data PDUFA fissata per il 19 giugno 2025. La richiesta segue la Designazione di Terapia Innovativa concessa nell'ottobre 2024.
Le NDA sono supportate da studi di Fase 3, PURPOSE 1 e PURPOSE 2. PURPOSE 1 ha mostrato una riduzione del rischio del 100% nelle donne cisgender, con zero infezioni nel gruppo lenacapavir. PURPOSE 2 ha dimostrato un'efficacia del 99,9% con una riduzione del rischio del 96% tra gli uomini cisgender e le persone di genere diverso, con solo due infezioni da HIV riportate.
Gilead ha anche presentato domande all'Agenzia Europea dei Medicinali per una revisione sotto valutazione accelerata, inclusa una domanda EU-M4all per facilitare la disponibilità nei paesi a basso e medio-basso reddito.
Gilead Sciences anunció que la FDA ha aceptado su Solicitud de Nuevo Medicamento para lenacapavir, un tratamiento inyectable para la prevención del VIH que se administra dos veces al año, bajo revisión prioritaria con una fecha PDUFA del 19 de junio de 2025. La solicitud sigue a la Designación de Terapia Innovadora otorgada en octubre de 2024.
Las NDA están respaldadas por ensayos de Fase 3, PURPOSE 1 y PURPOSE 2. PURPOSE 1 mostró una reducción del riesgo del 100% en mujeres cisgénero, con cero infecciones en el grupo de lenacapavir. PURPOSE 2 demostró una eficacia del 99,9% con una reducción del riesgo del 96% entre hombres cisgénero y personas de género diverso, con solo dos infecciones de VIH reportadas.
Gilead también ha presentado solicitudes a la Agencia Europea de Medicamentos para revisión bajo evaluación acelerada, incluyendo una solicitud EU-M4all para facilitar la disponibilidad en países de ingresos bajos y medios-bajos.
길리어드 사이언스는 FDA가 레나카파비르에 대한 신약 신청을 우선 심사로 수락했다고 발표했습니다. 이 약물은 연 2회 주사로 투여되는 HIV 예방 치료제이며, PDUFA 날짜는 2025년 6월 19일로 설정되어 있습니다. 이 신청은 2024년 10월에 부여된 혁신 치료제 지정에 따른 것입니다.
신약 신청은 3상 임상 시험인 PURPOSE 1과 PURPOSE 2에 의해 뒷받침됩니다. PURPOSE 1에서는 여성 cisgender 그룹에서 100%의 감염 위험 감소가 나타났으며, 레나카파비르 그룹에서는 감염이 없었습니다. PURPOSE 2에서는 cisgender 남성과 성별 다양성이 있는 사람들 사이에서 99.9%의 효능과 96%의 위험 감소가 나타났으며, HIV 감염이 단 두 건만 보고되었습니다.
길리어드는 또한 유럽 의약품청에 가속 평가를 위한 신청서를 제출했으며, 저소득 및 중저소득 국가에서의 이용 가능성을 촉진하기 위해 EU-M4all 신청도 포함되어 있습니다.
Gilead Sciences a annoncé que la FDA a accepté sa Demande de Nouveau Médicament pour lenacapavir, un traitement injectable de prévention du VIH à administrer deux fois par an, dans le cadre d'un examen prioritaire avec une date PDUFA fixée au 19 juin 2025. La demande fait suite à la désignation de thérapie innovante accordée en octobre 2024.
Les NDA sont soutenues par des essais de phase 3, PURPOSE 1 et PURPOSE 2. PURPOSE 1 a montré une réduction du risque de 100 % chez les femmes cisgenres, avec zéro infection dans le groupe lenacapavir. PURPOSE 2 a démontré une efficacité de 99,9 % avec une réduction du risque de 96 % chez les hommes cisgenres et les personnes de genre divers, avec seulement deux infections à VIH signalées.
Gilead a également soumis des demandes à l'Agence européenne des médicaments pour un examen dans le cadre d'une évaluation accélérée, y compris une demande EU-M4all pour faciliter la disponibilité dans les pays à faible et moyen revenu.
Gilead Sciences gab bekannt, dass die FDA ihren Antrag auf Zulassung eines neuen Arzneimittels für Lenacapavir, ein halbjährliches injizierbares HIV-Präventionsmittel, im Rahmen eines beschleunigten Verfahrens akzeptiert hat, mit einem PDUFA-Datum vom 19. Juni 2025. Der Antrag folgt auf die im Oktober 2024 erteilte Auszeichnung als Durchbruchtherapie.
Die NDAs werden durch die Phase-3-Studien PURPOSE 1 und PURPOSE 2 unterstützt. PURPOSE 1 zeigte eine Risikominderung von 100% bei cisgender Frauen, mit null Infektionen in der Lenacapavir-Gruppe. PURPOSE 2 demonstrierte eine Wirksamkeit von 99,9% mit einer Risikominderung von 96% bei cisgender Männern und geschlechtsdiversen Personen, wobei nur zwei HIV-Infektionen gemeldet wurden.
Gilead hat auch Anträge bei der Europäischen Arzneimittel-Agentur auf Überprüfung im Rahmen einer beschleunigten Bewertung eingereicht, einschließlich eines EU-M4all-Antrags zur Erleichterung der Verfügbarkeit in einkommensschwachen und niedrigen mittleren Einkommensländern.
- FDA granted Priority Review status, potentially expediting approval process
- PURPOSE 1 trial showed 100% risk reduction with zero infections
- PURPOSE 2 trial demonstrated 99.9% effectiveness with 96% risk reduction
- If approved, would be first twice-yearly HIV prevention option
- Received Breakthrough Therapy Designation in October 2024
- Product not yet approved in any market globally
- Two HIV infections occurred in PURPOSE 2 trial lenacapavir group
Insights
The FDA's acceptance of lenacapavir's NDA under Priority Review marks a potential paradigm shift in HIV prevention. The twice-yearly dosing represents a significant competitive advantage over current daily oral PrEP options, addressing one of the primary challenges in HIV prevention - medication adherence.
The clinical data is exceptionally robust, with PURPOSE 1 showing 100% risk reduction in cisgender women and PURPOSE 2 demonstrating 96% risk reduction in cisgender men and gender-diverse people. These efficacy rates surpass the historical benchmarks set by daily oral PrEP options.
The market implications are substantial. The global HIV PrEP market, currently dominated by daily oral medications, could see a major shift. Lenacapavir's dosing advantage could expand the addressable market by attracting individuals who have been hesitant to commit to daily medication. The dual regulatory strategy - FDA Priority Review and EMA Accelerated Assessment - suggests a coordinated global launch plan that could accelerate market penetration.
Gilead's access strategy, particularly the EU-M4all application, indicates a sophisticated commercial approach that balances premium market opportunities with broader global access. This could create a tiered pricing model that maximizes both market penetration and revenue potential.
The June 19, 2025 PDUFA date could mark the beginning of a new era in HIV prevention, potentially strengthening Gilead's dominant position in the HIV market. However, successful commercialization will require significant investment in manufacturing infrastructure for injectable formulations and healthcare provider education about administration protocols.
– If Approved, Lenacapavir Would Be the First and Only Twice-Yearly HIV Prevention Choice –
– FDA to Review Applications Under Priority Review, with a PDUFA Date of June 19, 2025 –
– Gilead Also Recently Submitted Applications for Lenacapavir for PrEP to the European Medicines Agency That Will Be Reviewed Under Accelerated Assessment Review Timeline –
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
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
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
There is currently no cure for HIV or AIDS.
About 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
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).
View source version on businesswire.com: https://www.businesswire.com/news/home/20250217805274/en/
Ashleigh Koss, Media
public_affairs@gilead.com
Jacquie Ross, Investors
investor_relations@gilead.com
Source: Gilead Sciences, Inc.
FAQ
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