Supplemental sNDA to the FDA for vutrisiran for the treatment of transthyretin amyloidosis with cardiomyopathy

Vutrisiran is the generic name for Amvuttra, which is currently approved by the  FDA for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults. As part of the submission, the Company utilized a Priority Review Voucher, which obligates the FDA to an accelerated review timeline.

“We are proud that with this first regulatory submission, we are a significant step closer to bringing vutrisiran to patients with ATTR amyloidosis with cardiomyopathy, which is a steadily progressive, debilitating, and ultimately fatal disease. HELIOS-B demonstrated rapid knockdown of TTR with vutrisiran and an improvement in death and cardiovascular events, as well as delays in disease progression, with vutrisiran as compared to placebo, with consistent effects across all pre-specified subgroups,” said Pushkal Garg, M.D., Chief Medical Officer, Alnylam. “We believe that, pending regulatory approval, vutrisiran has the potential to become a first-line therapy for ATTR amyloidosis with cardiomyopathy. We look forward to working with the FDA over the coming months on this application to bring this medicine to patients as rapidly as possible. Additional filings in other geographies are underway.”

The application to the FDA was based on positive results from HELIOS-B, a Phase III, randomized, double-blind, placebo-controlled multicenter global study in patients with ATTR-CM with substantial background use of other effective therapies. 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. The safety profile of vutrisiran in HELIOS-B was consistent with the established profile of the drug. In HELIOS-B, rates of adverse events (AEs), serious AEs, severe AEs, and AEs leading to study drug discontinuation were similar between the vutrisiran and placebo arms.