FDA issues CRL on Alnylam's Onpattro sNDA

The US Food and Drug Administration (FDA) has issued a complete response letter (CRL) in response to Alnylam Pharmaceuticals’ (Nasdaq: ALNY) supplemental New Drug Application (sNDA) for patisiran for the treatment of the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis. The news pushed the firm’s shares down more than 6% to $166.16 by mid-morning.

Trade named Onpattro, patisiran is approved by the FDA for the treatment of the polyneuropathy of hereditary ATTR amyloidosis in adults. The CRL does not pertain to, nor does it impact commercial availability of, Onpattro for this existing indication.

Onpattro, whose sales last year reached $558 million, won FDA approval back in 2018 for patients with polyneuropathy due to hereditary ATTR, and shortly after secured a European approval.