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Pharmaceuticals / Submissions and Approvals

FDA Awards Breakthrough Therapy Designation to Alnylam’s Givosiran

June 5, 2017

Alnylam Pharmaceuticals received a Breakthrough Therapy designation from the FDA for givosiran (ALN-AS1), an investigational RNAi therapeutic for the prevention of neurovisceral attacks in patients with acute hepatic porphyria.

Givosiran was previously granted PRIME designation by the European Medicines Agency. The company plans to initiate a Phase III clinical program late this year. The product has also been granted Orphan Drug designations in Europe and the U.S. for the treatment of acute hepatic porphyrias.

An ongoing placebo-controlled Phase I study of givosiran demonstrated initial reductions in both the number and frequency of porphyria attacks, as well as reductions in annualized hemin doses required in patients with acute intermittent porphyria, the most common and severe form of the disease.

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