Vutrisiran, sold under the brand name Amvuttra, is a
medication used for the treatment of the
polyneuropathy
Polyneuropathy ( poly- + neuro- + -pathy) is damage or disease affecting peripheral nerves (peripheral neuropathy) in roughly the same areas on both sides of the body, featuring weakness, numbness, and burning pain. It usually begins in the hand ...
of
hereditary transthyretin-mediated (hATTR) amyloidosis in adults.
It is a
small interfering RNA (siRNA) that interferes with the expression of the
transthyretin (TTR) gene.
Vutrisiran was approved for medical use in the United States in June 2022,
and in the European Union in September 2022.
History
The U.S.
Food and Drug Administration (FDA) granted the application for vutrisiran
orphan drug
An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases.
The assignment of ...
designation.
Society and culture
Legal status
On 21 July 2022, the
Committee for Medicinal Products for Human Use (CHMP) of the
European Medicines Agency
The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of medicinal products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products or Euro ...
(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.
[ Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.] Vutrisiran was approved for medical use in the European Union in September 2022.
[ Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.]
Names
Vutrisiran is the
international nonproprietary name (INN).
References
External links
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Orphan drugs
RNA interference
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