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RNAi Drug for Acute Hepatic Porphyria

The US Food and Drug Administration has approved the RNA interference drug givosiran (Alnylam Pharmaceuticals' Givlaari) to treat acute hepatic porphyria in adults, according to the agency.

Acute hepatic porphyria is an inherited disorder in which porphyrin accumulates during heme production and can lead to pain, paralysis, seizures and other symptoms, as MedPage Today reports. According to Alnylam, there are four types of acute hepatic porphyria, each of which stem from genetic alterations affecting enzymes in the liver's heme biosynthesis pathway. Givosiran is an RNAi drug that targets aminolevulinic acid synthase 1 mRNA to reduce its levels and the levels of toxins associated with attacks.

"Prior to today's approval, treatment options have only provided partial relief from the intense unremitting pain that characterizes these attacks," Richard Pazdur, the director of FDA's Oncology Center of Excellence and acting director of the Office of Oncologic Diseases says in a statement. "The drug approved today can treat this disease by helping to reduce the number of attacks that disrupt the lives of patients."

Reuters adds that the drug will cost about $575,000 per year.