This investigational ribonucleic acid interference (RNAi) therapeutic is aimed at treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM).
The FDA has set a prescription drug user fee act (PDUFA) action date goal of 23 March next year, facilitated by the company’s use of a priority review voucher. Currently, the US regulator does not plan to convene an advisory committee meeting to discuss the application.
The sNDA submission is supported by positive outcomes from the HELIOS-B global Phase III trial. This multicentre, randomised, double-blind, placebo-controlled trial involved 655 subjects with ATTR amyloidosis, either hereditary or wild-type, with cardiomyopathy.
Subjects were randomised to receive either vutrisiran 25mg or a placebo subcutaneously every three months for up to 36 months.
Following the double-blind treatment period, subjects had the option to continue receiving vutrisiran during an open-label extension period.
The safety and tolerability of vutrisiran were consistent with its known profile, even when administered alongside standard care treatments.
Alnylam Pharmaceuticals chief medical officer Pushkal Garg said: “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.
“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 US early next year.”
Vutrisiran, known under the brand name AMVUTTRA, is already FDA-approved for addressing the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in the adult population.
The potential approval of the therapy would mark it as the first US-approved treatment for both polyneuropathy and cardiomyopathy manifestations of ATTR amyloidosis.