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FDA grants orphan drug designation to ATX-F8-117 for hemophilia A
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The FDA granted orphan drug designation for ATX-F8-117 for the treatment of patients with hemophilia A, according to a press release from the drug’s manufacturer.
ATX-F8-117 (Apitope) — a preclinical product candidate — is designed to prevent or treat inhibitors in patients with hemophilia A who have inhibitors or who are at risk for developing Factor VIII inhibitors.
Hemophilia A is a genetic disorder that causes missing or defective Factor VIII, an essential blood clotting protein, according to the press release. Clinicians normally treat patients with hemophilia A with factor VIII inhibitors; however, because their immune systems have had low or no exposure to Factor VIII, patients often are not fully tolerant of replacement Factor VIII and up to 30% develop Factor VIII inhibitor antibodies.
No approved treatments are currently available to prevent or treat the development of inhibitors.
“Following the orphan drug designation by the European Medical Agency last year, we are very pleased to receive designation for the U.S. FDA for ATX-F8-117,” Keith Martin, PhD, CEO of Apitope, said in a press release. “These designations emphasize the need for an effective treatment for hemophilia A patients developing Factor VIII inhibitors that occur in approximately 30% of patients. This results in poor clotting of the blood, leading to severe health problems. This orphan drug designation follows extensive pre-clinical evaluation and we look forward to advancing a clinical development program for this important medical condition.”
The FDA’s Office of Orphan Products Development grants orphan drug designation to novel drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the United States.