FDA Approves Long-Acting Hemophilia B Treatment

The FDA approved Idelvion (coagulation factor IX [recombinant], albumin fusion protein; CSL Behring) for use in children and adults with hemophilia B. This is the first coagulation factor-albumin fusion protein product and the second factor IX fusion protein product that is modified to last longer in blood, to be approved in the US.

Factor IX is a naturally occurring clotting factor that is missing or defective in people with hemophilia B. This new treatment is used to replace factor IX.

Idelvion is indicated as needed for control and prevention of bleeding episodes, management of bleeding after surgery, and as a routine prophylaxis to reduce the frequency of bleeding episodes.

When used for prevention, coagulation factor IX (recombinant), albumin fusion protein potentially requires less frequent injections than unmodified factor IX.

Safety and efficacy were evaluated in two multicenter studies including 90 adults and pediatric patients with hemophilia B. The treatment was found effective in controlling bleeding episodes and managing bleeding after surgery. When used as a prophylaxis, there was a significant reduction in the rate of spontaneous bleeding episodes per year despite less frequent infusions.

There were no safety concerns identified in the study. The most common side effect observed was headache.