FDA grants Fast Track designation for vutrisiran to treat the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis.- Alnylam Pharma

Alnylam Pharmaceuticals, Inc. announced that the FDA has granted Fast Track designation to vutrisiran, an investigational therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. According to the FDA, Fast Track designation is designed to facilitate the development and expedite the review of drugs that treat serious conditions and fill an unmet medical need. With this designation, Alnylam will be eligible to submit a rolling New Drug Application for vutrisiran.

“Vutrisiran has demonstrated an encouraging safety profile in the Phase 1 study, with infrequent quarterly dosing with low-volume, subcutaneous administration which potentially reduces the burden of care for this progressive, life-threatening and multisystem disease. We are therefore pleased that the FDA has granted vutrisiran Fast Track designation,” said Rena Denoncourt, Vutrisiran Program Leader at Alnylam. “After completing enrollment earlier this year, we look forward to sharing topline results of the HELIOS-A Phase III study of vutrisiran in early 2021.