Advertisement Apitope gets FDA orphan drug status for ATX-F8-117 to treat Haemophilia A patients - Pharmaceutical Business review
Pharmaceutical Business review is using cookies

ContinueLearn More
Close

Apitope gets FDA orphan drug status for ATX-F8-117 to treat Haemophilia A patients

Belgium-based Apitope has received orphan drug designation from the US Food and Drug Administration (FDA) for its pre-clinical product candidate, ATX-F8-117, to prevent or treat inhibitors in haemophilia A patients with inhibitors or at the risk of producing inhibitors.

Haemophilia A is a rare chronic bleeding disorder, which leads to inadequate clotting of blood in response to any type of injury or surgery and about 30% of these patients develop Factor VIII inhibitor antibodies.

According to the company 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.

The company through its patented discovery platform has 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: "Following the Orphan Drug Designation by the European Medical Agency last year, we are very pleased to receive designation from the US FDA for ATX-F8-117.

"These designations emphasise the need for an effective treatment for haemophilia A patients developing Factor VIII inhibitors that occurs in approximately 30 per cent of patients.

"This results in poor clotting of the blood leading to severe health issues. This orphan drug designation follows extensive pre-clinical evaluation and we look forward to advancing a clinical development programme for this important medical condition."

At present, there are few therapies available to help patients with inhibitors making the Apitope approach potentially life changing for patients.