Apitope Receives Orphan Designation in the European Union for ATX-F8-117 for the treatment of Haemophilia A
Apitope 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.
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Antibodies are specialized molecules of our immune system that can specifically recognize and neutralize pathogens or foreign substances. Antibody research in biotech and pharma has recognized this natural defense potential and is working intensively to make it therapeutically useful. From monoclonal antibodies used against cancer or autoimmune diseases to antibody-drug conjugates that specifically transport drugs to disease cells - the possibilities are enormous
Topic world Antibodies
Antibodies are specialized molecules of our immune system that can specifically recognize and neutralize pathogens or foreign substances. Antibody research in biotech and pharma has recognized this natural defense potential and is working intensively to make it therapeutically useful. From monoclonal antibodies used against cancer or autoimmune diseases to antibody-drug conjugates that specifically transport drugs to disease cells - the possibilities are enormous