News Feature | December 3, 2014

Apitope's Hemophilia A Drug Awarded Orphan Status In EU

By Estel Grace Masangkay

Pharmaceutical firm and immunotherapy developer Apitope announced that the European Medicines Agency’s Committee for Orphan Medicinal Products (EMA COMP) has awarded orphan medicinal product designation to the company’s ATX-F8-117 for the treatment of bleeding disorder hemophilia A.

ATX-F8-117 is derived from Factor VIII and is being developed as a peptide therapy to prevent inhibitor development in hemophilia A patients treated with Factor VIII. Factor VIII is an important blood-clotting protein that is rendered dysfunctional or is missing in patients with hemophilia A due to a genetic defect.

Hemophilia A is a rare and chronic bleeding disorder in which the blood fails to clot following injury or surgery. Patients are typically treated with Factor VIII to facilitate normal clotting of the blood. However, due to the fact that patients’ bodies had low or zero exposure to the protein, most are not tolerant to the replacement Factor VIII. Up to 30 percent of patients treated with the protein replacement therapy develop Factor VIII antibodies.

Dr. Keith Martin, CEO of Apitope International, said, “We are very pleased to receive Orphan Medicinal Product Designation by the EMA for ATX-F8-117, which underlines the need for an effective treatment for patients with hemophilia A. ATX-F8-117 is currently in preclinical development for the treatment of Factor VIII inhibitors which develop in approximately 30 percent of patients and results in poor clotting leading to severe health issues for patients. More specifically, the potential impact of this product could be an important step in the fight to help hemophilia A patients who cannot benefit fully from Factor VIII replacement therapy."

In May, Apitope launched the preclinical development of ATX-F8-117 to treat Factor VIII intolerance in patients with hemophilia A.

The company is also developing another peptide therapy called ATX-GD-459 for the treatment of Graves' disease. Apitope launched the preclinical trial of ATX-GD-459 in June.