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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

DateArticle
Jun 13, 2022Approval FDA Approves Amvuttra (vutrisiran) for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults
Apr  4, 2022Alnylam Announces 3-Month Extension of Review Period for New Drug Application for Vutrisiran
Jan 21, 2022Alnylam Presents Positive 18-Month Results from HELIOS-A Phase 3 Study of Investigational Vutrisiran in Patients with hATTR Amyloidosis with Polyneuropathy
Jun 24, 2021Alnylam Announces U.S. Food and Drug Administration Acceptance of New Drug Application for Investigational Vutrisiran for the Treatment of the Polyneuropathy of Hereditary ATTR Amyloidosis
Apr 19, 2021Alnylam Presents Positive Results from HELIOS-A Phase 3 Study of Investigational Vutrisiran
Jan  7, 2021Alnylam Reports Positive Topline Results from HELIOS-A Phase 3 Study of Vutrisiran in Patients with hATTR Amyloidosis with Polyneuropathy
Apr 14, 2020Alnylam Receives Fast Track Designation for Vutrisiran for the Treatment of the Polyneuropathy of hATTR Amyloidosis

Further information

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