The U.S. Food and Drug Administration (FDA) has approved Givlaari (givosiran) to treat acute hepatic porphyria, a rare genetic disorder that causes a buildup of porphyrins in the body.

Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research said, “This buildup can cause acute attacks, known as porphyria attacks, which can lead to severe pain and paralysis, respiratory failure, seizures and mental status changes. These attacks occur suddenly and can produce permanent neurological damage and death.”

Givosiran is an RNA interference (RNAi) medicine that targets aminolevulinic acid synthase 1 (ALAS1), an enzyme that accumulates to toxic levels in the liver of people with acute hepatic porphyria.

In the video clip below,  Akshay Vaishnaw, MD, PhD, President, Research and Development at Alnylam Pharmaceuticals, explains how RNAi medications can work in condition like acute hepatic porphyria.

 

 

The approval of givosiran was largely based on a Phase III clinical trial involving 94 patients with acute hepatic porphyria. In the study, the givosiran-treated group experience 70% fewer porphyria attached compared the the placebo-treated group.

Common adverse events are nausea and injection site reactions.

 

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