Home » FDA Approves First Treatment for Inherited Rare Disease
FDA Approves First Treatment for Inherited Rare Disease
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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