|Drug class||Small interfering RNA (siRNA)|
|Main uses||Hepatic porphyria|
|Side effects||Pain at the site of injection, nausea, tiredness|
|Typical dose||2.5 mg/kg q 1 month|
|Chemical and physical data|
|Molar mass||16300.42 g·mol−1|
|3D model (JSmol)|
Common side effects include pain at the site of injection, nausea, and tiredness. Other side effects may include anaphylaxis, kidney problems, and liver problems. It is a small interfering RNA (siRNA) that decreases the amount of the enzyme aminolevulinic acid synthase 1 (ALAS1) and therefore decreases the production of heme.
Givosiran was approved for medical use in the United States in 2019 and Europe in 2020. In the United Kingdom a 189 mg vial costs the NHS about £4200 as of 2021. This amount in the United States costs about 40,700 USD.
It is generally given at a dose of 2.5 mg/kg.
The U.S. Food and Drug Administration (FDA) granted the application for givosiran breakthrough therapy designation, priority review designation, and orphan drug designation. The FDA granted the approval of Givlaari to Alnylam Pharmaceuticals. The FDA considers it to be a first-in-class medication.
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- "Drug Trials Snapshots: Givlaari". U.S. Food and Drug Administration (FDA). 20 November 2019. Retrieved 24 January 2020. This article incorporates text from this source, which is in the public domain.
- "New Drug Therapy Approvals 2019". U.S. Food and Drug Administration. 31 December 2019. Retrieved 15 September 2020.