Hemophilia B Treatment, Idelvion, Approved for Use in Japan

Japan’s Ministry of Health, Labor and Welfare has approved CSL Behring‘s Idelvion (albutrepenonacog alfa) for the prevention of bleeding tendency in patients with hemophilia B.

Hemophilia B, a bleeding condition that mainly affects males, is characterized by a deficient or defective factor IX protein, and is treated with intravenous infusion of factor IX. However, the stability of this factor, measured by means of half-life, is poor.

Idelvion is a long-acting albumin fusion protein linking recombinant coagulation factor IX with recombinant albumin. Its approval in Japan covers its use in both children and adults tor regular prophylaxis, to prevent or reduce the frequency of bleeding episodes; for on-demand control; and for the management of bleeding around the time of surgery.

The approval was based on data from the PROLONG-9FP clinical development program, composed of five Phase 1 through Phase 3 efficacy and safety trials. Results showed that Idelvion delivers high-level protection with up to 14-day dosing in appropriate patients, with a median annualized spontaneous bleeding rate (AsBR) of 0.00 for all treatment intervals. This was observed in both children and adults (ages 1 to 61) with hemophilia B (factor IX levels ≤ 2%).

Data was published in the journal Blood, in the study, “Long acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in hemophilia B: results of a phase 3 trial.”

Idelvion was approved in 2016 in the U.S, the European Union, and Canada.

“IDELVION provides excellent bleeding control by maintaining a high factor IX activity level over a prolonged period of time,” Dr. Andrew Cuthbertson, chief scientific officer and R&D director for CSL Limited, said in a recent press release. “IDELVION delivers on CSL’s 100 year promise to develop and provide innovative specialty biotherapies that patients need and want.  We look forward to bringingIDELVION to the Japan market and are particularly excited about the positive impact this long-acting therapy can have on the lives of patients with hemophilia B as CSL enters its next century.”

CSL recently presented six posters highlighting Idelvion, including interim results on a Phase 3b safety and efficacy study, at the XXXII International Congress of the World Federation of Hemophilia (WFH) 2016 .