The FDA rejected a supplemental new drug application for patisiran to treat cardiomyopathy of wild-type transthyretin-mediated or hereditary transthyretin-mediated amyloidosis, citing insufficient evidence of clinical meaningfulness.
In a complete response letter issued by FDA, the agency stated that it did not identify any clinical safety, study conduct, drug quality or manufacturing issues for patisiran (Onpattro, Alnylam). As a result of the FDA’s decision, Alnylam stated in a press release that it will no longer pursue an expanded indication for patisiran in the U.S.; however, it