Long-Acting Hemophilia Drug Approved by FDA

The FDA has approved a long-acting fusion protein drug for the treatment of hemophilia B in children and adults.

Idelvion [Coagulation factor IX (recombinant), albumin Fusion Protein] is novel long acting albumin fusion protein that links recombinant coagulation factor IX with recombinant albumin for treating patients with hemophilia B. It is also the first and only factor IX therapy that gives high level protection for up to 14-day dosing.

“Idelvion has the potential to significantly impact the treatment of hemophilia B as it maintains factor IX activity levels above 5% over a prolonged period of time,” said Chief Scientific Officer and R&D Director at CSL Limited, Andrew Cuthbertson. “This provides excellent bleeding control. Idelvion is the first product from our innovative recombinant factor development program to receive FDA approval. We are proud to add this new therapy to our growing portfolio of bleeding disorder products, and are particularly excited about the positive impact treatment with Idelvion can have on the well-being of patients with hemophilia B.”

Long Acting Treatment of Hemophilia B

The approval was based off results from the PROLONG-9FP clinical development program, which includes phase 1-3 open label, multicenter studies that evaluated the safety and efficacy of Idelvion.

The results of the studies showed that the dosing interval was achieved while maintaining high levels of factor activity above 5% (and below 50%) over 14 days at 75 IU/kg. The most common adverse reactions were headaches.

For on-demand therapy, the data showed 94% of bleeds were controlled with 1 infusion and 99% were controlled with 1 or 2 infusions.

“The approval of this long-acting recombinant factor IX therapy for hemophilia B is vital, as physicians need more options to help their patients effectively and safely manage their bleeding disorder,” said lead investigator of the PROLONG-9FP clinical development program, Elena Santagostino, MD, PhD. “As the only recombinant factor IX therapy offering up to 14-day dosing, Idelvion helps patients maintain factor IX activity levels over a long period of time. This provides them with greater freedom from frequent infusions which is an important attribute for patients who require a prophylactic regimen but don’t want treatment to disrupt their active lives.”