FDA Approves Expanded Indication for IXINITY to Include All Patients With Hemophilia B

The US Food and Drug Administration (FDA) has approved a supplemental Biologics License Application (sBLA) for the recombinant coagulation factor IX agent, IXINITY^®, according to a release from the developer, Medexus Pharmaceuticals.

IXINITY^® is used to prevent and manage bleeding episodes in patients with hemophilia B. The sBLA expanded the indication for IXINITY^® to include pediatric patients under 12 years of age. With the expanded indication, IXINITY^® is now approved in all patients with hemophilia B.

“We are pleased to secure approval for IXINITY^® for use in pediatric patients, a population that comprises approximately one-third of individuals with hemophilia B in the United States,” said Ken d’Entremont, Chief Executive Officer of Medexus. “We hope the greater availability of IXINITY^® will help ease the burden on children and families who face the challenge of managing hemophilia B and allow children living with this lifelong condition to enjoy more active lives.”

The sBLA was supported by data from a phase III/IV study on IXINITY^® as prophylaxis for bleeding episodes in previously treated pediatric patients with severe or moderately severe hemophilia B. The study’s authors reported low annualized bleeding rates, effective management of bleeding episodes, and pharmacokinetics and safety consistent with adult populations.

One of the principal investigators of the study, Johnny Mahlangu, BSc, MBBCh, MMed, FCPath, noted that, while dose adjustment may be necessary in patients aged 12 years and under, “The [study’s] results thus provide further validation of the clinical utility of IXINITY^® as a treatment for all people living with hemophilia B.”