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NOVEMBER 21, 2019

FDA approves Givlaari for Rare Acute Hepatic Porphyria

By Marie Rosenthal

The FDA granted approval to givosiran (Givlaari, Alnylam) to treat adults with acute hepatic porphyria (AHP), a genetic disorder resulting in the buildup of toxic porphyrin molecules, which are formed during the production of heme.

Approval of givosiran was received in less than four months after acceptance of the New Drug Application, and was based on positive results from the phase 3 ENVISION, a multinational, randomized, double-blind, placebo-controlled trial in 94 patients