Announced on October 9, the decision to issue a CRL is based on the FDA's belief the clinical meaningfulness of patisiran’s treatment effects for the cardiomyopathy of ATTR amyloidosis had not been established.
A multipart, phase 1 trial provides evidence demonstrating the safety of zilbesarian, an RNA interference agent, which appeared to have a favorable safety profile among adult patients with hypertension.
Data from the open-label extension period of the APOLLO-B trial provide new insight into the benefits of patisiran in patients with ATTR amyloidosis with cardiomyopathy.