FDA Grants Orphan Status for ATX-F8-117 for the treatment of Haemophilia A patients in the US
Commenting on the designation, Dr Keith Martin, CEO 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.”
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