The U.S. Food and Drug Administration on Thursday approved Alnylam Pharmaceuticals Inc.’s supplemental application for Amvuttra (vutrisiran) in adults with cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis, or ATTR-CM, to reduce cardiovascular mortality, cardiovascular hospitalizations, and urgent heart failure visits.

The approval expands the indication for Amvuttra, which now becomes the first and only therapeutic approved by the FDA for ATTR-CM and the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.

ATTR-CM affects approximately 150,000 people in the U.S. and over 300,000 people worldwide.

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This approval is based on the HELIOS-B Phase 3 clinical trial. The trial achieved statistical significance compared to placebo on all 10 pre-specified primary and secondary endpoints.

In the overall population, Amvuttra reduced the risk of all-cause mortality and recurrent cardiovascular (CV) events by 28% during the double-blind treatment period of up to 36 months.

Mortality in this population was significantly reduced by 36% through 42 months in a pre-specified secondary endpoint analysis.

In the monotherapy population, Amvuttra reduced the risk of ACM and recurrent CV events by 33% and the risk of mortality by 35% through 42 months.

Amvuttra in hATTR-PN is covered by insurers for around 99% of patients, with the majority paying nothing out-of-pocket. Similar broad coverage and out-of-pocket costs are expected in ATTR-CM, given comparable payer dynamics and the clinical value demonstrated in the HELIOS-B clinical trial.

Marketing authorization applications based on HELIOS-B data are currently under review by several global health agencies including the European Medicines Agency, the Brazilian Health Regulatory Agency, and the Japanese Pharmaceuticals and Medical Devices Agency.

Alnylam remains on track to proceed with additional global regulatory submissions for vutrisiran in 2025.

In 2024, Amvuttra sales reached $970.45 million, up from $557.84 million a year ago.

Price Action: ALNY stock is up 8.46% at $274.98 at the last check Friday.

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