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Trade names | Givlaari |
AHFS/Drugs.com | Monograph |
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Routes of administration | Subcutaneous |
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Chemical and physical data | |
Formula | C524H694F16N173O316P43S6 |
Molar mass | 16300.42 g·mol−1 |
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Givosiran, sold under the brand name Givlaari, is a medication used for the treatment of adults with acute hepatic porphyria. [3] [6] [7] [8] Givosiran is a small interfering RNA (siRNA) directed towards delta-aminolevulinate synthase 1 (ALAS1), [9] an important enzyme in the production of heme.
The most common side effects include nausea and injection site reactions. [8]
The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication. [10]
Givosiran is indicated 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). [3] [7]
In November 2019, givosiran was approved in the United States for the treatment of adults with acute hepatic porphyria (AHP). [7] [11]
Efficacy was evaluated in ENVISION (NCT03338816), a randomized, double‑blind, placebo‑controlled, multinational trial enrolling 94 participants with acute hepatic porphyria. [8] Participants were randomized (1:1) to receive once monthly subcutaneous injections of givosiran 2.5 mg/kg or placebo during a six‑month double‑blind period. [8] The performance of givosiran was measured by the rate of porphyria attacks that required hospitalizations, urgent health care visits or intravenous infusion of hemin at home. [7] Participants who received givosiran experienced 70% fewer porphyria attacks compared to patients receiving a placebo. [7]
The U.S. Food and Drug Administration (FDA) granted the application for givosiran breakthrough therapy designation, priority review designation, and orphan drug designation. [7] [8] The FDA granted the approval of Givlaari to Alnylam Pharmaceuticals. [7]
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