Breakthrough Therapy designation has been granted by the US Food and Drug Administration for givosiran (ALN-AS1), an investigational RNAi therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) for the prophylaxis of attacks in patients with acute hepatic porphyria (AHP).
The drug is under development by Alnylam Pharmaceuticals (Nasdaq: ALNY), whose shares gained 2.14% to $65.46 by close of trading yesterday following the announcement.
Breakthrough Therapy designation is granted to expedite the development and review of new drugs that treat serious or life-threatening diseases where preliminary clinical evidence exists in support of substantial benefit over available therapies.
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