Alnylam Pharmaceuticals Gets FDA Breakthrough Therapy Designation

Alnylam Pharmaceuticals Gets FDA Breakthrough Therapy Designation

Alnylam Pharmaceuticals shares rose 2% in pre-market trade Wednesday after the Food and Drug Administration granted it breakthrough therapy designation for givosiran, an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 for the prophylaxis of attacks in patients with acute hepatic porphyria. Acute hepatic porphyrias is a rare metabolic disorder caused by deficiency within the liver of one of the eight enzymes responsible for heme biosynthesis. “We believe givosiran could […]

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