sNDA submitted to the FDA for Onpattro for the treatment of the cardiomyopathy of ATTR amyloidosis.
Alnylam Pharmaceuticals, Inc. announced the submission of its supplemental New Drug Application (sNDA) to the FDA for patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy
Patisiran is the established name for Onpattro which is currently approved by the FDA for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults.
The application to the FDA was based on positive results from APOLLO-B, a Phase III, randomized, double-blind, placebo-controlled multicenter global study that demonstrated the effects of patisiran on functional capacity and quality of life in patients with ATTR amyloidosis with cardiomyopathy.
The safety profile in APOLLO-B was consistent with what was observed in APOLLO and in postmarketing use of Onpattro. In APOLLO-B, the majority of adverse events (AEs) were mild or moderate in severity. The 12-month results from the study were presented at the 18th International Symposium on Amyloidosis (ISA) on September 8, 2022.
Learning Zones
The Learning Zones are an educational resource for healthcare professionals that provide medical information on the epidemiology, pathophysiology and burden of disease, as well as diagnostic techniques and treatment regimens.
1 mins