Alnylam Pharmaceuticals has submitted a supplemental New Drug Application (sNDA) to the US Food and Drug Administration (FDA) for patisiran, which is being investigated for the treatment of transthyretin amyloidosis (ATTR) with cardiomyopathy.

“We are excited by today’s announcement as the submission of our sNDA brings us one step closer to making patisiran available to patients with ATTR amyloidosis with cardiomyopathy. Patisiran has the potential to improve the functional capacity and quality of life of patients with ATTR amyloidosis with cardiomyopathy, an increasingly recognized cause of heart failure for which there are limited treatment options,” said Rena Denoncourt, vice president and TTR franchise lead at Alnylam Pharmaceuticals.

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“The positive results of the APOLLO-B Phase 3 study of patisiran validate the therapeutic hypothesis that TTR silencing by an RNAi therapeutic may be an effective approach to treating cardiomyopathy of both wild-type and hereditary ATTR amyloidosis. This is an important milestone as we work to build an industry-leading franchise for the treatment of ATTR amyloidosis.”

Alnylam Pharmaceuticals presented 12-month results from the APOLLO-B study at the 18th International Symposium on Amyloidosis showing positive effects of patisiran on patients’ functional capacity, health status, and quality of life.

The safety profile obtained in the study was encouraging and consistent with what was observed in the APOLLO trial and in postmarketing use of Onpattro®, which is currently approved by the FDA for the treatment of polyneuropathy associated with hereditary ATTR (hATTR) in adults. In general, patisiran was well-tolerated, with most adverse events being mild or moderate in severity.

Reference

Alnylam submits supplemental New Drug Application (sNDA) to U.S. Food and Drug Administration (FDA) for ONPATTRO® (patisiran) for the treatment of the cardiomyopathy of ATTR amyloidosis. News release. Alnylam Pharmaceuticals, Inc; December 8, 2022.