Alnylam Pharmaceuticals, Inc., a leading RNA interference (RNAi) therapeutics company, recently announced that it has submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for vutrisiran, an investigational RNAi therapeutic being developed for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM).
Vutrisiran, marketed under the name AMVUTTRA, has already received FDA approval for treating polyneuropathy associated with hereditary ATTR amyloidosis in adults.
To expedite the review process, Alnylam utilized a Priority Review Voucher, ensuring a faster evaluation by the FDA.
The sNDA submission is backed by promising data from the HELIOS-B trial, a Phase 3, randomized, double-blind, placebo-controlled global study involving patients with ATTR-CM who were also receiving other effective treatments.
The HELIOS-B study showed that vutrisiran had significant positive impacts on reducing mortality rates and cardiovascular events, while also enhancing functional capacity and quality of life for patients with ATTR-CM.
The safety of vutrisiran in the HELIOS-B trial corresponded with its known safety profile. The incidence of adverse events (AEs), serious AEs, severe AEs, and discontinuations due to AEs were similar between patients taking vutrisiran and those on a placebo.