CSL Behring has disclosed a temporary global stockout of its gene therapy for hemophilia B, potentially delaying treatment for some patients. The company communicated the supply issue directly to the patient community through a letter.
Hemophilia B is a rare bleeding disorder caused by deficiency of clotting factor IX, affecting approximately 1 in 25,000 males worldwide. Gene therapies represent a breakthrough treatment option for patients with severe forms of the condition, offering the potential for long-term or permanent relief from regular factor replacement therapy.