The U.S. Food and Drug Administration has expanded the orphan drug designation for Alnylam Pharmaceuticals Inc.'s drug patisiran.
Patisiran's orphan drug designation now covers the treatment of transthyretin-mediated amyloidosis, an inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene. TTR protein is produced primarily in the liver and is normally a carrier of vitamin A.
The drug was previously given the designation to treat familial amyloidotic polyneuropathy.
Also, the European Medicines Agency has granted accelerated assessment for patisiran.
The company intends to file a new drug application with the FDA and a marketing authorization application in the EU by the end of 2017.
The drug will be commercialized by Alnylam in the U.S., Canada, and Western Europe, while Sanofi's Sanofi Genzyme will commercialize the drug in the rest of the world, including certain Central and Eastern European countries of the EU.