- Patisiran Marketing Authorization Application to be reviewed under accelerated assessment
Paris and Cambridge, Mass. – December 18, 2017 – Sanofi Genzyme, the specialty care global business unit of Sanofi, and Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of adults with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). Patisiran was previously granted accelerated assessment by the EMA, potentially reducing the EMA’s evaluation time from 210 to 150 days.
“The MAA submission for patisiran represents another important milestone for Alnylam and a critical step toward bringing RNAi therapeutics to people living with hATTR amyloidosis,” said Eric Green, Vice President and General Manager of the TTR program at Alnylam. “Based on the results of the APOLLO study, we believe patisiran has the potential to become the standard of care for the treatment of hATTR amyloidosis. We look forward to working with the EMA and the Committee for Medicinal Products for Human Use (CHMP) during the review process.”
“People with hATTR amyloidosis have limited treatment options,” said Rand Sutherland, M.D., Therapeutic Area Head, Rare Diseases Development at Sanofi. “With this MAA submission, we are one step closer to making patisiran available in Europe and executing on our shared vision to bring this RNAi treatment to patients globally.”
U.S and other regulatory submissions
Alnylam announced completion of the submission of a New Drug Application with the U.S. Food and Drug Administration (FDA) on December 12, 2017. Sanofi Genzyme is currently preparing regulatory filings for patisiran in Japan, Brazil and other countries, with submissions expected to begin 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, including certain Central and Eastern European countries of the European Union.
Patisiran has not been approved by the FDA, EMA, or any other regulatory authority and no conclusions can or should be drawn regarding the safety or effectiveness of this investigational therapeutic.