Amvuttra FDA Approval History
Last updated by Judith Stewart, BPharm on March 24, 2025.
FDA Approved: Yes (First approved June 13, 2022)
Brand name: Amvuttra
Generic name: vutrisiran
Dosage form: Injection
Company: Alnylam Pharmaceuticals, Inc.
Treatment for: Hereditary Amyloidosis, Cardiomyopathy of Transthyretin-Mediated Amyloidosis
Amvuttra (vutrisiran) is an RNAi therapeutic for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis and cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis.
- Amvuttra is indicated for the treatment of:
- the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.
- the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality, cardiovascular hospitalizations and urgent heart failure visits. - Transthyretin amyloidosis (ATTR) is a progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body. There are two different forms of ATTR - hereditary ATTR (hATTR) which is caused by a TTR gene variant and wild-type ATTR (wtATTR) which occurs without a TTR gene variant. The amyloid deposits of ATTR accumulate in various parts of the body, including the nerves and the heart, and patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease.
- FDA approval for hATTR-PN was based on results of the HELIOS-A Phase 3 study which demonstrated that Amvuttra significantly improved polyneuropathy with over 50% of patients experiencing improvement in neuropathy impairment relative to baseline. Approval for ATTR-CM was based on the HELIOS-B Phase 3 study which achieved statistical significance for Amvuttra compared to placebo on all 10 pre-specified primary and secondary endpoints.
- Amvuttra is administered by a healthcare professional via subcutaneous injection every 3 months.
- Warnings and precautions associated with Amvuttra include reduced serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) is recommended.
- Common adverse reactions (≥5%) include pain in extremity, arthralgia, dyspnea, and decreased vitamin A levels.
Development timeline for Amvuttra
Further information
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