trending Market Intelligence /marketintelligence/en/news-insights/trending/jDF8R-xY4uPZXpnS5O4BmQ2 content esgSubNav
In This List

US FDA expands orphan designation for Alnylam's patisiran


According to Market Intelligence, December 2022


Insight Weekly: Layoffs swell; energy efficiency PE deals defy downturn; 2023 global risk themes


Insight Weekly: Energy crisis cripples Europe; i-bank incomes rise; US holiday sales outlook


Japan M&A By the Numbers: Q3 2022

US FDA expands orphan designation for Alnylam's patisiran

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.