Alnylam Announces Positive Outcome of FDA Advisory Committee Meeting on Patisiran for the Treatment of the Cardiomyopathy of ATTR Amyloidosis

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced the positive outcome of the U.S. Food and Drug Administration’s (FDA) Cardiovascular and Renal Drugs Advisory Committee (CRDAC) meeting to discuss the supplemental New Drug Application (sNDA) for patisiran, an investigational RNAi therapeutic in development for the treatment of the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis. The CRDAC voted 9:3 that the benefits of patisiran outweigh its risks for the treatment of the cardiomyopathy of ATTR amyloidosis.

“We are grateful for the CRDAC’s thoughtful review and discussion, and thank the patients, physicians and advocacy community who shared their valuable insights today,” said Pushkal Garg, M.D., Chief Medical Officer at Alnylam. “The positive outcome of today’s meeting is supported by the efficacy and safety data observed in the APOLLO-B Phase 3 study, and is another step toward bringing patients with the cardiomyopathy of ATTR amyloidosis a novel treatment option that addresses the underlying cause of disease and has the potential to meaningfully benefit patients’ functional capacity and quality of life. We look forward to continuing to work with the FDA as they complete their review of our sNDA.”

ATTR amyloidosis is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the heart, resulting in cardiomyopathy and heart failure. The cardiac manifestations associated with ATTR amyloidosis can have a devastating impact on patients’ lives and treatment options today are limited, with many patients continuing to progress on, or unable to access, current standard-of-care.

The positive decision by the FDA’s CRDAC panel of independent experts was based on a discussion of the data supporting the sNDA for patisiran, which include positive results from the APOLLO-B Phase 3 study that demonstrate favorable effects on functional capacity and health status and quality of life in patients with ATTR amyloidosis with cardiomyopathy relative to placebo, as measured by the 6-Minute Walk Test (6-MWT) and the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) score, respectively.