Posted by Michael Wonder on 17 Nov 2017
Alnylam initiates rolling submission of new drug application to U.S. FDA for patisiran for the treatment of hereditary ATTR amyloidosis
16 November 2017 - Company plans to complete submission by year end.
Alnylam Pharmaceuticals today announced the company has initiated submission of a rolling new drug application to the U.S. FDA for patisiran, an investigational RNAi therapeutic targeting transthyretin, for the treatment of hereditary ATTR amyloidosis. The rolling submission allows completed portions of an NDA to be reviewed by the FDA on an ongoing basis. Alnylam has submitted the non-clinical and chemistry, manufacturing and controls components and expects to submit the final clinical data component by the end of the year. Alnylam has also requested a priority review of the application which, if granted, could result in a six-month review process. Patisiran previously received fast track designation from the FDA.
Alnylam, in alliance with Sanofi Genzyme, plans to submit a marketing authorisation application to the EMA around year end. Sanofi Genzyme is currently preparing for regulatory filings for patisiran in Japan, Brazil and other countries, to begin in the first half of 2018.
