Regulator OKs Takeda Korea’s acquired hemophilia A treatment

Takeda Korea said Wednesday that it received approval for Obizur, a treatment for bleeding in adult patients with acquired hemophilia A (AHA), from the Ministry of Food and Drug Safety.
AHA is a disease characterized by the development of autoimmune antibodies against blood coagulation factor 8, resulting in bleeding-related complications.

It is a rare disease affecting about one in a million people per year, and patients with AHA tend to have more fatal bleeding events than those with congenital hemophilia A.

Most patients are elderly, with a median age of 64-78, and severe or life-threatening bleeding occurs in 70-90 percent of patients. The mortality rate can increase by more than 20 percent if advanced age or underlying malignancy is involved.

Unlike congenital hemophilia A, it is difficult to determine the severity of the disease by the concentration of the blood coagulation factor 8 in the blood.

As a result, it is necessary to consider clinical bleeding symptoms for prompt diagnosis and bleeding control.

Unlike existing bypass factor agents, Obizur is the first treatment to replace factor 8 for AHA indications.

The recombinant product removes the B-domain from human-like porcine factor 8. As autoimmune antibodies do not readily recognize the treatment, Obizur can replace inactivated human factor 8 to help blood clot and control bleeding.

Because of such a mechanism, Takeda Korea stressed that Obizur is the only treatment for AHA that reliably monitors blood factor 8 levels using standard assays, allowing for personalized dosing.

“We are very pleased and excited to announce the approval of Obizur in Korea, which will provide Korean AHA patients with a new treatment option that can replace the existing bypass agents,” said Kim Na-kyung, head of the hemophilia business division at Takeda Korea.