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FDA grants fast track designation to DTX101 for hemophilia B
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The FDA granted fast track designation to DTX101, a gene therapy product in development for the treatment of patients with hemophilia B, according to a press release from the agent’s manufacturer.
DTX101 (Dimension Therapeutics) is designed to deliver blood clotting Factor IX gene expression in patients with this condition. The current standard treatment requires patients to undergo chronic replacement of Factor IX protein via IV infusion.
A multicenter phase 1/phase 2 trial to evaluate DTX101 in adults with moderate to severe hemophilia B is expected to begin by the end of this year.
More than 28,000 people worldwide — including an estimated 4,000 people in the United States — have hemophilia B, according to World Federation of Hemophilia estimates.
“We are very pleased to achieve this additional milestone for our DTX101 program, following the FDA’s recent acceptance of Dimension’s investigational new drug application and granting of orphan drug designation for our lead candidate,” Annalisa Jenkins, MBBS, MRCP, CEO of Dimension Therapeutics, said in the press release. “This latest development provides further momentum.”