Givosiran
Givosiran, sold under the brand name Givlaari, is for the treatment of adults with acute hepatic porphyria, a genetic disorder resulting in the buildup of toxic porphyrin molecules which are formed during the production of heme (which helps bind oxygen in the blood).[1][2]
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Trade names | Givlaari |
Routes of administration | Subcutaneous injection |
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Formula | C524H694F16N173O316P43S6 |
Molar mass | 16300.42 g·mol−1 |
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History
The U.S. Food and Drug Administration (FDA) granted the application for givosiran breakthrough therapy designation, priority review designation, and orphan drug designation.[1] The FDA granted the approval of Givlaari to Alnylam Pharmaceuticals.[1]
References
- "FDA approves first treatment for inherited rare disease". U.S. Food and Drug Administration (FDA) (Press release). 20 November 2019. Archived from the original on 21 November 2019. Retrieved 20 November 2019.
This article incorporates text from this source, which is in the public domain. - "FDA approves givosiran for acute hepatic porphyria". U.S. Food and Drug Administration (FDA) (Press release). 20 November 2019. Archived from the original on 21 November 2019. Retrieved 20 November 2019.
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