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# FDA Accepts Alnylam NDA for Patisiran
- URL: https://www.fdaweb.com/fda-accepts-alnylam-nda-for-patisiran/
- Published: 2018-02-01T12:00:00.000Z
- Updated: 2026-09-14T23:18:50.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5140665

FDA has accepted for priority review an Alnylam Pharmaceuticals NDA for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for treating hereditary ATTR (hATTR) amyloidosis. The inherited, progressively debilitating, and often fatal disease is caused by mutations in the TTR gene. TTR protein is produced primarily in the liver and is normally a carrier of vitamin A. Mutations in TTR cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as the peripheral nerves and heart, resulting in intractable peripheral sensory neuropathy, autonomic neuropathy, and cardiomyopathy, according to the company. The agency has set a user fee review action target date of 8/11.

Alnylam says patisiran is designed to silence specific messenger RNA, potentially blocking the production of TTR protein before it is made. “This may help to enable the clearance of TTR amyloid deposits in peripheral tissues and potentially restore function to these tissues,” it says.