GeneVentiv Therapeutics, a pre-clinical gene therapy company, announced that the FDA has granted Orphan Drug Designation (ODD) to GENV-HEM for the treatment of Hemophilia A or B with or without inhibitors.
"I am pleased to announce our Orphan Drug Designation for GENV-HEM from the FDA. With no approved gene therapies for hemophilia patients with inhibitors, we are dedicated to driving GENV-HEM forward as the first gene therapy for both hemophilia A or B with or without inhibitors," said Damon Race, CEO of GeneVentiv.
The FDA Office of Orphan Products Development grants orphan designation for novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. Orphan designation qualifies the sponsor of the drug for various development incentives of the Orphan Drug Act, including a seven-year period of U.S. marketing exclusivity, tax credits for clinical research costs, clinical research trial design assistance, the ability to apply for annual grant funding and waiver of Prescription Drug User Fee Act filing fees.
Hemophilia is an inherited bleeding disorder caused by a deficiency of the functional clotting factor. GENV-HEM is an Adeno-Associated Virus containing a transgene encoding for activated factor V (FVa) variant. Within the coagulation pathway, FVa functions downstream of FVIII and FIX as a co-factor of FXa to promote hemostasis. A single infusion of GENV–HEM would result in endogenous expression of FVa, improving hemostasis in patients with either FIX or FVIII deficiencies independent of the presence of any respective clotting factor inhibitors. GENV-HEM has the potential to offer multiple benefits for severe hemophilia patients including multi-year disease ameliorating hemostatic phenotype.
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