Alnylam Pharmaceuticals FDA Accepts Application for Vutrisiran to Treat ATTR Amyloidosis with Cardiomyopathy
Tuesday, November 26, 2024
Alnylam Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for vutrisiran.
AMVUTTRA is an RNAi-based therapy administered via quarterly subcutaneous injections. The treatment is designed to reduce TTR protein levels, addressing the underlying mechanism of ATTR. Currently approved in over 15 countries for hATTR-PN in adults, it is also under development for cardiomyopathy forms of the disease.
ATTR amyloidosis is a progressive and often fatal disease caused by misfolded TTR proteins accumulating in tissues such as nerves and the heart. It manifests in hereditary (hATTR) and wild-type (wtATTR) forms, affecting up to 350,000 people globally.
RNAi is a natural process of gene silencing, offering a novel approach to disease treatment. By targeting messenger RNA (mRNA), RNAi therapies prevent the production of disease-causing proteins. This breakthrough has paved the way for innovative treatments like vutrisiran, transforming care for patients with genetic and other serious conditions.
The investigational treatment targets transthyretin amyloidosis with cardiomyopathy (ATTR-CM). With the use of a Priority Review Voucher, the FDA has set a target decision date of 23 March 2025 under the Prescription Drug User Fee Act (PDUFA).
The agency has also indicated that it does not plan to convene an advisory committee meeting for the review.
Vutrisiran, marketed under the name AMVUTTRA®, is already approved in the United States for hereditary transthyretin-mediated amyloidosis (hATTR-PN) with polyneuropathy in adults.
By reducing both mutant and wild-type transthyretin (TTR) protein levels, vutrisiran addresses the root cause of ATTR. If approved, it would be the first treatment in the U.S. to manage both polyneuropathy and cardiomyopathy manifestations of the condition.
The FDA’s decision follows positive data from the HELIOS-B Phase 3 clinical trial, which evaluated vutrisiran in patients with ATTR-CM. The global study, conducted as a double-blind, placebo-controlled trial, demonstrated significant improvements in cardiovascular outcomes, disease progression, survival, and quality of life.
These effects were observed in patients already receiving standard care treatments. Vutrisiran also showed a favourable safety and tolerability profile consistent with previous findings.
Full results were presented at the European Society of Cardiology Congress and published in The New England Journal of Medicine in August 2024.
Source: alnylam.com
