Orphan Drug Designation for rVIIa-FP for Hemophilia A and Hemophilia B

CSL Behring announced that the FDA has granted its request for orphan drug designation for recombinant fusion protein linking coagulation factor VIIa with albumin (rVIIa-FP) for treatment and prophylaxis of bleeding episodes in patients with congenital hemophilia and inhibitors to coagulation factor VIII or IX.

Hemophilia is a congenital bleeding disorder characterized by prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs. The disease is caused by deficient or defective blood coagulation proteins known as factor VIII or IX. In Hemophilia A, or classic hemophilia, the clotting factor VIII is either deficient or defective. Hemophilia B is characterized by deficient or defective factor IX. The recommended treatment for patients who are factor deficient is to treat by replacement-factor therapy. A complication in some patients is the development of inhibitory antibodies (inhibitors) to FVIII or FIX which renders replacement therapy ineffective. This can occur in up to 25% of hemophilia A patients and around 5% of hemophilia B patients. One treatment option for these patients is recombinant activated factor VII (called a bypassing agent) which can be used to achieve hemostasis without the need for factor VIII or IX.

Preclinical studies have shown that rVIIa-FP has favorable increases in half-life and may offer patients the opportunity to be treated less frequently than with currently available product.

For more information, call (877) 236-4423 or visit www.cslbehring.com.