FDA Approves First Treatment for Inherited Factor X Deficiency
The US Food and Drug Administration (FDA) has approved Coagadex, a significant breakthrough for managing inherited factor X deficiency. This rare blood clotting disorder affects approximately 1 in 500,000 to 1 million people worldwide. The approval comes after extensive research and clinical trials, offering hope to those living with this condition.
Factor X deficiency, also known as Stuart-Prower factor deficiency, is caused by insufficient factor X protein in the blood. This leads to an increased risk of excessive bleeding. Diagnosis involves a blood test called a factor X assay, which measures the activity of factor X. Other tests, such as prothrombin time (PT) test, partial thromboplastin time, and thrombin time, may also be used.
The disorder can be inherited or acquired. Inherited factor X deficiency is relatively rare, while acquired factor X deficiency is even less common, often caused by conditions like severe liver disease, vitamin K deficiency, or certain medications. The outlook for those with factor X deficiency varies depending on the type and severity of the condition, with potential risks including severe bleeding and complications during pregnancy. Treatment for inherited factor X deficiency may involve blood transfusions or medication like tranexamic acid.
The FDA approval of Coagadex marks a significant milestone in the management of inherited factor X deficiency. This therapy, the first of its kind, offers a new option for those living with this rare condition. While there is no cure for inherited factor X deficiency, the availability of Coagadex provides a promising avenue for improved treatment and quality of life.
