Amvuttra FDA Approval History
Last updated by Judith Stewart, BPharm on July 1, 2022.
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
Amvuttra (vutrisiran) is an RNAi therapeutic for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.
- Hereditary transthyretin-mediated (hATTR) amyloidosis is an inherited, progressively debilitating, and fatal disease caused by mutations in the TTR gene. The mutations cause abnormal amyloid proteins to accumulate, causing polyneuropathy.
- Amvuttra is a double‑stranded small interfering RNA (siRNA) that targets mutant and wild‑type transthyretin (TTR) messenger RNA (mRNA) to block the production of the TTR protein.
- FDA approval is 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.
- Amvuttra is administered via subcutaneous injection every 3 months.
- Common adverse reactions include arthralgia, dyspnea, and decreased serum levels of vitamin A. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking Amvuttra.
Development timeline for Amvuttra
Further information
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