U.S. Food and Drug Administration (FDA) grants Alnylam Breakthrough Therapy Designation (BTD) for patisiran for the treatment of hereditary ATTR (hATTR) amyloidosis with polyneuropathy.
Alnylam Pharmaceuticals, Inc., the leading RNAi therapeutics company, announced the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of adults with hereditary transthyretin-mediated ATTR amyloidosis (hATTR amyloidosis) with polyneuropathy.
Breakthrough Therapy Designation is intended to expedite the development and review of drugs for the treatment of serious or life-threatening conditions based on clinical evidence indicating that the drug demonstrates substantial improvement on clinically significant endpoints over available therapy.
lnylam has initiated a rolling New Drug Application (NDA) for patisiran and expects the last submission by the end of 2017. Patisiran previously received Fast Track Designation in the U.S., and was recently granted accelerated assessment by the European Medicines Agency. Alnylam, in alliance with Sanofi Genzyme, intends to file a marketing authorization application in the European Union around year-end.
“Hereditary ATTR amyloidosis is an aggressive, rapidly progressing, debilitating and fatal disease, and the need for new treatment options is urgent. The robust evidence provided by the APOLLO Phase 3 study demonstrated the potential of investigational patisiran to transform the lives of people with hATTR amyloidosis,” said Eric Green, vice president and general manager of the TTR program. “Breakthrough Therapy Designation enables us to work closely with the FDA on the review of the NDA to potentially bring patisiran to patients living with this devastating disease as quickly as possible.”
Pending regulatory approvals, Alnylam will commercialize patisiran in the U.S., Canada, and Western Europe, with Sanofi Genzyme commercializing the product in the rest of the world.
(Source: Alnylam Pharmaceuticals, Inc.)
Filed Under: Drug Discovery