Posted by Michael Wonder on 13 Dec 2017
Alnylam completes submission of new drug application to U.S. FDA for patisiran for the treatment of hereditary ATTR amyloidosis
12 December 2017 - Patisiran could become the first in a new class of medicines known as RNAi therapeutics.
Alnylam Pharmaceuticals announced today the completion of the rolling submission of a new drug application to the U.S. FDA for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of adults with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis).
The rolling submission began on 15 November 2017 with the non-clinical and chemistry, manufacturing and controls components submitted, and now submission of the clinical data completes the filing. Alnylam has requested priority review for the application which, if granted, could result in a six-month review process. Patisiran previously received fast track and breakthrough therapy designations from the FDA, and recently received an expanded Orphan Drug Designation for ATTR amyloidosis.
