CHMP recommends Onpattro to treat hereditary transthyretin-mediated amyloidosis.- Alnylam Pharma.

Alnylam Pharmaceuticals, Inc.announced that the Committee for Medicinal Products for Human Use (CHMP) has adopted a Positive Opinion recommending marketing authorization of patisiran for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adults with stage 1 or stage 2 polyneuropathy. If approved by the European Commission (EC), the medicine will be commercialized under the brand name Onpattro.

The CHMP positive opinion is based on the evaluation of the effects of patisiran in patients with hATTR amyloidosis and its safety profile as demonstrated in the APOLLO Phase III study. The SmPC recommended by the CHMP includes data from APOLLO primary and secondary endpoints, as well as exploratory cardiac endpoints. The results of the APOLLO study were published 5 July, 2018 in The New England Journal of Medicine (NEJM).