Sanofi Genzyme, Alnylam Announce MAA Submission

Sanofi Genzyme and Alnylam Pharmaceuticals announced 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.

“People with hATTR amyloidosis have limited treatment options,” said Rand Sutherland, M.D., therapeutic area head of 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.”

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.