|Main uses||Polyneuropathy in hereditary transthyretin-mediated (hATTR) amyloidosis|
|Side effects||Joint pain, shortness of breath, vitamin A deficiency|
|Typical dose||25 mg q 3 months|
Vutrisiran, sold under the brand name Amvuttra, is a medication used to treat polyneuropathy in hereditary transthyretin-mediated (hATTR) amyloidosis. It is used in adults who are still able to walk. It is used by injection under the skin.
Common side effects include joint pain, shortness of breath, and vitamin A deficiency. Vitamin A supplements are recommended. It is a small interfering RNA (siRNA) that decreases expression of the transthyretin (TTR) gene.
It is generally used at a dose of 25 mg every 3 months.
Society and culture
On 21 July 2022, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Amvuttra, intended for treatment of hereditary transthyretin-mediated (hATTR) amyloidosis. Amvuttra was designated as an orphan medicinal product on 25 May 2018. The applicant for this medicinal product is Alnylam Netherlands B.V. Vutrisiran was approved for medical use in the European Union in September 2022.
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