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 become a transformative treatment for patients with this devastating and potentially life-threatening disease,” said Jeff Miller, general manager of the company’s Givosiran program. “Accordingly, we look forward to rapidly advancing this program in collaboration with global regulatory authorities, having also received PRIME designation from the European Medicines Agency earlier this year. We plan to initiate the phase 3 clinical program with givosiran in late 2017.”