- The U.S. Food and Drug Administration (FDA) accepted for review Alnylam Pharmaceuticals' ( NASDAQ: ALNY ) application seeking expanded approval of patisiran to treat cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis.
- The FDA is expected to make a decision on the supplemental new drug application (sNDA) by Oct. 8.
- Alnylam said that the FDA is planning to hold an advisory committee meeting to discuss the application.
- The filing was backed by data from a phase 3 trial called APOLLO-B.
- Patisiran is sold under the name Onpattro, which is approved in the U.S. to treat polyneuropathy of hereditary ATTR amyloidosis in adults.
- "ATTR amyloidosis with cardiomyopathy is an increasingly recognized cause of heart failure for which there are limited treatment options," said Rena Denoncourt, vice president, TTR Franchise Lead.
- ATTR amyloidosis is a rare, progressive, debilitating disease caused by misfolded transthyretin (TTR) proteins which build up as amyloid fibrils in multiple tissues including the nerves, heart, and gastrointestinal tract, the company noted.
For further details see:
Alnylam gets FDA review for expanded use of rare disease drug Onpattro