GENV-HEM, an investigational treatment for both hemophilia A and B, has received the Orphan Drug Designation from the US Food and Drug Administration (FDA).

The announcement was made by GeneVentiv Therapeutics, the preclinical gene therapy company developing the treatment, in a press release. 

Hemophilia is characterized by low levels of clotting factors in the blood, leading to spontaneous bleeds. Hemophilia A is caused by a mutation in the gene encoding one such factor, factor VIII (FVIII), while hemophilia B is caused by a mutation in the gene that encodes factor IX (FIX).

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The disease is treated with factor replacement, however, some patients develop inhibitors or antibodies against the administered clotting factor.

GENV-HEM is a gene therapy for activated factor V, which functions downstream of FVIII and FIX to promote hemostasis. The gene that encodes activated factor V is carried to the body via an adeno-associated virus. It is hoped that following treatment with GENV-HEM, the body will start to produce activated factor V, thereby improving hemostasis regardless of whether FVIII, FIX, or any factor inhibitors are present. 

Valder Arruda, MD, PhD, the chairman of GeneVentiv’s scientific advisory board, stated, “This unique approach focused on the ‘bypass’ strategy . . . has the potential to simplify the treatment of severe hemophilia and to provide an innovative intervention for hemophilia A with refractory inhibitors to FVIII or those hemophilia B with inhibitors to FIX with contraindication of FIX protein/transgene exposure FIX, which are certainly challenging clinical scenarios.”

Orphan Drug Designation is granted to novel drugs or biological products that treat a disease or condition affecting fewer than 200,000 patients in the United States. The designation allows the developer of the drug or biological product to have access to various incentives, such as a 7-year period of US marketing exclusivity, tax credits for clinical research costs, and assistance with clinical research trial design.


GeneVentiv receives Orphan Drug Designation (ODD) for GENV-HEM for the treatment of hemophilia A or B with or without inhibitors. News release. GeneVentiv Therapeutics; November 1, 2021.