FDA Approves Gene Therapy for Hemophilia A

Severe cases of the bleeding disorder can be life-threatening.

The U.S. Food and Drug Administration (FDA) has granted approval for Roctavian, gene therapy for adults with severe hemophilia A who do not have pre-existing antibodies to “adeno-associated virus serotype 5.” Hemophilia A is a rare genetic bleeding disorder caused by a deficiency of clotting factor VIII (FVIII).

“Hereditary hemophilia A is a potentially serious bleeding disorder. Severe cases of hemophilia A can cause life-threatening health issues due to increased risk of uncontrolled bleeding,” said FDA’s Center for Biologics Evaluation and Research Director Peter Marks. He added that this “approval represents an important advance in providing treatment options for patients with this bleeding disorder, and treatment with gene therapy may reduce the need for ongoing routine therapy.”

As the Lord Leads, Pray with Us…

  • For Director Marks as he oversees the Center for Biologics Evaluations and Research.
  • For FDA scientists as they evaluate data and studies regarding the efficacy of proposed therapies.
  • For those suffering from Hemophilia A to get the care they need.

Sources: Food and Drug Administration


Back to top