The U.S. Food and Drug Administration granted orphan drug designation to Alnylam Pharmaceuticals Inc.'s ALN-TTRsc02 for the treatment of a neurodegenerative disease known as transthyretin-mediated amyloidosis.
The designation is granted to medicines being developed to treat rare diseases or disorders that affect fewer than 200,000 people in the U.S.
The Cambridge, Mass.-based company's treatment is also designated as an orphan medicinal product in the EU.
