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Home » FDA Approves First Treatment for Inherited Rare Disease
FDA Approves First Treatment for Inherited Rare Disease
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December 3, 2019
Alnylam Pharmaceuticals nabbed FDA approval for Givlaari for treatment of adults with acute hepatic porphyria, a genetic disorder resulting in the buildup of toxic porphyrin molecules.
The FDA granted the approval based on the results of a clinical trial of 94 patients with acute hepatic porphyria. The trial showed patients who received Givlaari (givosarin) experienced 70 percent fewer porphyria attacks compared to patients receiving a placebo.
Givlaari is the first FDA approved treatment for acute hepatic porphyria. It previously received breakthrough therapy, priority review and orphan drug designations.
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