EMA Grants Alnylam Accelerated Assessment of Patisiran

Alnylam Pharmaceuticals announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has granted an accelerated assessment for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR (hATTR) amyloidosis. The EMA awards an accelerated assessment to medicines deemed to be of major public health interest and therapeutic innovation, and the award is designed to bring new treatments to patients more quickly. Alnylam intends to file a marketing authorization application (MAA) in the European Union (EU) at year-end 2017. Accelerated assessment potentially provides a reduced review timeline from 210 to 150 days once the MAA is filed and validated.

"We are pleased the CHMP has granted accelerated assessment for patisiran and believe this underscores the urgent need to improve outcomes for patients living with hATTR amyloidosis," said Eric Green, Vice President and General Manager of the TTR program. "The results from the APOLLO Phase 3 study in hATTR amyloidosis patients with polyneuropathy provide evidence that patisiran has the potential to improve neurologic impairment, quality of life, and cardiac parameters. With this accelerated assessment award from the EMA, we hope to be able to make this promising treatment option available to patients in Europe sooner."

Alnylam plans to file a New Drug Application for patisiran in the United States by the end of 2017, where Fast Track Designation has been granted. Sanofi Genzyme is currently preparing for regulatory filings for patisiran in Japan, Brazil and other countries, beginning in the first half of 2018. 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.