Alnylam’s RNAi therapeutic Onpattro has been approved for use in Europe to treat Hereditary Transthyretin-Mediated (hATTR) Amyloidosis (hATTR amyloidosis) in adults with Stage 1 or Stage 2 polyneuropathy.
Sanofi Genzyme and Alnylam Pharmaceuticals have submitted a marketing application to the European Medicines Agency for patisiran, an investigational RNAi therapeutic targeting transthyretin for the treatment of adults with hereditary transthyretin-mediated amyloidosis.