FDA rejects Alnylam's patisiran sNDA for ATTR cardiomyopathy; company abandons U.S. expansion

ALNYLAM PHARMACEUTICALS, INC.

FDA issued Complete Response Letter citing insufficient evidence of clinical meaningfulness for patisiran in cardiomyopathy of ATTR amyloidosis.
No safety, study conduct, drug quality or manufacturing issues; existing ONPATTRO polyneuropathy indication unchanged.
Alnylam will no longer pursue expanded U.S. indication for patisiran; focus shifts to vutrisiran (HELIOS-B Phase 3) and ALN-TTRsc04.
FDA advisory committee had voted 9:3 that benefits outweigh risks, but FDA still issued CRL.
Company to host investor call at 8:30 a.m. ET on Oct 9, 2023 to discuss the CRL.

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