Alnylam Announces U.S. Food and Drug Administration Acceptance of Supplemental New Drug Application for Vutrisiran for the Treatment of ATTR Amyloidosis with Cardiomyopathy
Alnylam Pharmaceuticals (ALNY) announced FDA acceptance of their supplemental New Drug Application (sNDA) for vutrisiran to treat ATTR amyloidosis with cardiomyopathy (ATTR-CM). The FDA set a PDUFA date of March 23, 2025. The application is based on positive results from the HELIOS-B Phase 3 study, which showed favorable effects on cardiovascular outcomes, survival, and quality of life in ATTR-CM patients. If approved, vutrisiran would become the first treatment approved in the U.S. for both polyneuropathy manifestations of hATTR and cardiomyopathy manifestations of ATTR amyloidosis.
Alnylam Pharmaceuticals (ALNY) ha annunciato l'accettazione da parte della FDA della loro richiesta supplementare di autorizzazione di un nuovo farmaco (sNDA) per vutrisiran, destinato al trattamento dell'amiloidosi ATTR con cardiomiopatia (ATTR-CM). La FDA ha fissato una data PDUFA per il 23 marzo 2025. La domanda si basa su risultati positivi provenienti dallo studio di Fase 3 HELIOS-B, che ha mostrato effetti favorevoli sugli esiti cardiovascolari, sulla sopravvivenza e sulla qualità della vita nei pazienti con ATTR-CM. Se approvato, vutrisiran diventerà il primo trattamento approvato negli Stati Uniti per entrambe le manifestazioni di polineuropatia della hATTR e le manifestazioni di cardiomiopatia dell'amiloidosi ATTR.
Alnylam Pharmaceuticals (ALNY) anunció la aceptación por parte de la FDA de su solicitud suplementaria de nuevo fármaco (sNDA) para el vutrisiran, destinado al tratamiento de la amiloidosis ATTR con cardiomiopatía (ATTR-CM). La FDA estableció una fecha PDUFA para el 23 de marzo de 2025. La solicitud se basa en resultados positivos del estudio de Fase 3 HELIOS-B, que mostró efectos favorables en los resultados cardiovasculares, la supervivencia y la calidad de vida en pacientes con ATTR-CM. Si se aprueba, el vutrisiran se convertiría en el primer tratamiento aprobado en EE. UU. tanto para las manifestaciones de polineuropatía de hATTR como para las manifestaciones de cardiomiopatía de la amiloidosis ATTR.
Alnylam Pharmaceuticals (ALNY)는 ATTR 심근병증(ATTR-CM) 치료를 위한 vutrisiran의 보충 신약 신청서(sNDA)가 FDA에 의해 승인되었음을 발표했습니다. FDA는 2025년 3월 23일로 PDUFA 일자를 설정했습니다. 이 신청서는 ATTR-CM 환자에서 심혈관 결과, 생존 및 삶의 질에 대한 긍정적인 영향을 보여준 HELIOS-B 3상 연구의 긍정적인 결과를 기반으로 하고 있습니다. 승인될 경우, vutrisiran은 hATTR의 다발신경병증 및 ATTR 아밀로이드증의 심근병증 두 가지 증상에 대해 미국에서 승인된 최초의 치료제가 될 것입니다.
Alnylam Pharmaceuticals (ALNY) a annoncé l'acceptation par la FDA de sa demande de nouveau médicament complémentaire (sNDA) pour le vutrisiran, destiné au traitement de l'amyloïdose ATTR avec cardiomyopathie (ATTR-CM). La FDA a fixé une date PDUFA au 23 mars 2025. La demande repose sur des résultats positifs provenant de l'étude de phase 3 HELIOS-B, qui a montré des effets favorables sur les résultats cardiovasculaires, la survie et la qualité de vie des patients atteints d'ATTR-CM. Si elle est approuvée, le vutrisiran deviendra le premier traitement approuvé aux États-Unis pour les manifestations de polyneuropathie de la hATTR et les manifestations de cardiomyopathie de l'amyloïdose ATTR.
Alnylam Pharmaceuticals (ALNY) gab bekannt, dass die FDA ihren Antrag auf ergänzende Neue Arzneimittelgenehmigung (sNDA) für vutrisiran zur Behandlung der ATTR-Amyloidose mit Kardiomyopathie (ATTR-CM) angenommen hat. Die FDA legte ein PDUFA-Datum für den 23. März 2025 fest. Der Antrag basiert auf positiven Ergebnissen aus der HELIOS-B-Phase-3-Studie, die günstige Auswirkungen auf kardiovaskuläre Ergebnisse, Überleben und Lebensqualität bei ATTR-CM-Patienten zeigte. Wenn genehmigt, würde vutrisiran die erste Behandlung in den USA werden, die für die Manifestationen von Polyneuropathie bei hATTR sowie für die Manifestationen von Kardiomyopathie bei ATTR-Amyloidose zugelassen ist.
- FDA acceptance of sNDA for expanded indication could significantly increase market opportunity
- Positive Phase 3 HELIOS-B trial results showing improved cardiovascular outcomes and survival
- Potential to become first treatment approved for both polyneuropathy and cardiomyopathy manifestations
- No advisory committee meeting planned, potentially streamlining approval process
- Extended timeline with PDUFA date set for March 2025
- Current treatment shows adverse reactions including pain in extremity (15%) and arthralgia (11%)
Insights
The FDA acceptance of vutrisiran's sNDA for ATTR-CM represents a significant milestone for Alnylam. The March 23, 2025 PDUFA date was expedited through a Priority Review Voucher, indicating the company's strategic urgency to expand the drug's market potential.
The HELIOS-B trial results demonstrated compelling efficacy in improving cardiovascular outcomes, survival and quality of life. Most notably, these benefits were achieved on top of existing standard care treatments, suggesting vutrisiran could become a valuable addition to the current treatment paradigm.
If approved, vutrisiran would gain a significant competitive advantage as the first therapeutic approved for both polyneuropathy and cardiomyopathy manifestations of ATTR amyloidosis. This dual indication could substantially expand the drug's market opportunity and strengthen Alnylam's position in the ATTR space.
The expansion into ATTR-CM represents a substantial market opportunity for Alnylam. The ATTR-CM patient population is significantly larger than the current hATTR-PN indication, potentially multiplying the drug's revenue potential. The absence of a planned advisory committee meeting suggests the FDA's confidence in the data package, potentially streamlining the approval process.
The strong efficacy data from HELIOS-B, published in the prestigious New England Journal of Medicine, adds credibility and could support rapid market adoption if approved. The established safety profile and convenient administration could help drive market penetration, particularly given the unmet need in ATTR-CM treatment.
− Prescription Drug User Fee Act (PDUFA) Date Set for March 23, 2025 −
Vutrisiran is the generic name for AMVUTTRA®, which is currently approved by the FDA for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. By rapidly knocking down both mutant and wild‑type transthyretin (TTR), vutrisiran addresses the underlying cause of transthyretin amyloidosis (ATTR). If approved, vutrisiran would become the first therapeutic approved in the
“We are pleased that the FDA has accepted our sNDA for vutrisiran for the treatment of ATTR with cardiomyopathy – a steadily progressing, debilitating and ultimately fatal disease,” said Pushkal Garg, M.D., Chief Medical Officer of Alnylam. “In HELIOS-B, treatment with vutrisiran improved cardiovascular outcomes, survival, disease progression and quality of life, as compared to placebo, in a population reflective of today’s patients on substantial background treatment. We look forward to working with the FDA to support their review of the application and bring vutrisiran to patients with ATTR-CM in the
The supplemental application to the FDA was based on positive results from HELIOS-B, a randomized, double-blind, placebo-controlled multicenter global Phase 3 study in patients with ATTR-CM. The study demonstrated favorable effects of vutrisiran on outcomes of death and cardiovascular events, functional capacity and quality of life in patients with ATTR-CM. These treatment effects were seen on top of substantial background standard of care treatments. Vutrisiran demonstrated encouraging safety and tolerability consistent with the established profile of the drug. Detailed results from the study were presented at the European Society of Cardiology Congress and simultaneously published in The New England Journal of Medicine on August 30, 2024.
AMVUTTRA® (vutrisiran)
Indication
AMVUTTRA is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Important Safety Information
Reduced Serum Vitamin A Levels and Recommended Supplementation
AMVUTTRA treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking AMVUTTRA. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with AMVUTTRA, as serum vitamin A levels do not reflect the total vitamin A in the body.
Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).
Adverse Reactions
The most common adverse reactions that occurred in patients treated with AMVUTTRA were pain in extremity (
For additional information about AMVUTTRA, please see the full
About AMVUTTRA® (vutrisiran)
AMVUTTRA® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of mutant and wild-type transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. Administered quarterly via subcutaneous injection, AMVUTTRA is approved and marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults. Vutrisiran is also in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), which encompasses both wild-type and hereditary forms of the disease. For more information about AMVUTTRA, including the full
About ATTR
Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant and impacts an estimated 200,000 – 300,000 people worldwide.
About RNAi
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors that encode for disease-causing or disease pathway proteins – thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.
About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® (patisiran), AMVUTTRA® (vutrisiran), GIVLAARI® (givosiran), OXLUMO® (lumasiran), and Leqvio® (inclisiran), which is being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in
Alnylam Forward-Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects including, without limitation, Alnylam’s expectations regarding the safety and efficacy of vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy, including the safety, tolerability and favorable effects of vutrisiran on outcomes of death and cardiovascular events, functional capacity and quality of life in patients with ATTR-CM; whether the FDA will convene an advisory committee in connection with the FDA’s review of Alnylam’s supplemental application for approval of vutrisiran for the treatment of patients with ATTR-CM; the potential for vutrisiran to obtain FDA approval for the treatment of patients with ATTR-CM, and the anticipated timing of such FDA approval; and the potential for Alnylam to achieve its Alnylam P5x25 vision of becoming a leading biopharma company should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: Alnylam’s ability to successfully execute on its “Alnylam P5x25” strategy; Alnylam’s ability to successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates, including vutrisiran; actions or advice of regulatory agencies and Alnylam’s ability to obtain regulatory approval for its product candidates, including vutrisiran, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam’s approved products globally; and any delays, interruptions or failures in the manufacture and supply of Alnylam’s product candidates or its marketed products; as well as those risks more fully discussed in the “Risk Factors” filed with Alnylam’s 2023 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q and in its other SEC filings. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.
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Alnylam Pharmaceuticals, Inc.
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(Investors and Media)
+1-617-682-4340
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(Investors)
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Source: Alnylam Pharmaceuticals, Inc.
FAQ
When is the PDUFA date for Alnylam's (ALNY) vutrisiran sNDA for ATTR-CM?
What were the key results from Alnylam's (ALNY) HELIOS-B trial for vutrisiran?
What is the current FDA-approved indication for Alnylam's (ALNY) AMVUTTRA (vutrisiran)?
