EMA committee grants orphan designation for Apitope’s ATX-F8-117 to treat haemophilia A

Haemophilia A is a rare chronic bleeding disorder that leads to inadequate clotting of the blood in response to any type of injury or surgery.

Patients with haemophilia A are normally treated with Factor VIII to help with the clotting of their blood.

The company along with 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.

Apitope CEO Dr Keith Martin 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 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 haemophilia A patients who cannot benefit fully from Factor VIII replacement therapy."

Based in Belgium and the UK, the drug discovery and development firm is focused on disease-modifying treatments for patients with autoimmune and allergic diseases.

The company’s discovery platform enables selection of potential disease-modifying peptide therapies for the autoimmune/allergic disease of interest; and has already generated a pipeline of seven programs in clinical and preclinical development, of which the lead program in multiple sclerosis is partnered with Merck Serono.