ALNY - Alnylam gets FDA review for expanded use of rare disease drug Onpattro

• 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.

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