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Alnylam submits US FDA application for hATTR amyloidosis drug

Alnylam Pharmaceuticals Inc. completed its submission of a new drug application to the U.S. Food and Drug Administration for patisiran to treat adults with hereditary transthyretin-mediated amyloidosis, or hATTR amyloidosis.

The company requested priority review for the application, which could result in a six-month review process.

Patisiran has previously received fast-track and breakthrough therapy designations from the FDA and recently received an expanded orphan drug designation for ATTR amyloidosis. The European Medicines Agency has also granted accelerated assessment to the therapy.

The company, in collaboration with Sanofi unit Sanofi Genzyme, intends to file a marketing authorization application in the EU around 2017-end. Sanofi Genzyme is preparing for regulatory filings for the drug in Japan, Brazil and other countries, to commence in the first half of 2018.

Alnylam will commercialize patisiran in the U.S., Canada and western Europe, while Sanofi Genzyme will commercialize the product in the rest of the world, including certain central and eastern European countries.

HATTR amyloidosis is an inherited, progressively debilitating and often fatal disease caused by mutations in the transthyretin gene. Transthyretin protein is produced primarily in the liver and is normally a carrier of vitamin A. The disease affects about 50,000 people worldwide.