Apitope, a drug discovery and development company focussed on disease-modifying treatments for patients with autoimmune and allergic diseases, announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has granted orphan medicinal product designation to ATX-F8-117 for the treatment of haemophilia A.

Haemophilia A is a rare chronic bleeding disorder which leads to inadequate clotting of the blood in response to any type of injury or surgery. It is a genetic disorder that causes missing or defective Factor VIII, an essential blood-clotting protein. Haemophilia A patients are normally treated with Factor VIII to help with the clotting of their blood. However, since these patients’ immune systems have had no or low exposure to normal Factor VIII, they are often not fully tolerant to the replacement Factor VIII used to treat their condition. As a consequence, up to 30 per cent of these patients develop Factor VIII inhibitor antibodies.

The development of these antibodies is the most serious complication that significantly limits the treatment of this disorder as well as increasing the cost burden. Apitope has, through its patented discovery platform, completed the research work to confirm that the two peptides in ATX-F8-117, derived from Factor VIII, have the potential to treat and prevent inhibitor development in haemophilia A patients treated with Factor VIII. Currently, there are few therapies available to help patients with inhibitors making the Apitope approach potentially life changing for patients.

Commenting on the designation, Dr Keith Martin, chief executive officer, 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 haemophilia A. ATX-F8-117 is currently in preclinical development for the treatment of Factor VIII inhibitors which develop in approximately 30 per cent 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 haemophilia A patients who cannot benefit fully from Factor VIII replacement therapy.”