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.