Accelerated EMA assessment for patisiran in hereditary ATTR amyloidosis

The European Medicines Agency has accepted the Marketing Authorization Application (MAA) and initiated its review for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of hereditary ATTR (hATTR) amyloidosis.

The filing of the MAA by US RNAi therapy specialist Alnylam Pharmaceuticals (Nasdaq: ALNY) was previously announced on December 18, 2017, and patisiran was previously granted accelerated assessment by the EMA, potentially reducing the Agency’s evaluation time from 210 to 150 days.

The safety and efficacy of patisiran have not been evaluated by the US Food and Drug Administration or any other health authority, although the FDA granted the drug coveted Breakthrough Therapy designation last November, resulting in Alnylam’s shares hitting a historical high. Alnylam announced completion of the submission of a New Drug Application with the US FDA on December 12, 2017.