The U.S. Food and Drug Administration (FDA) has approved a supplemental Biologics License Application for IXINITY® (Medexus), a recombinant factor replacement therapy to treat patients with hemophilia B. 

The new approval expands the product indication to include the on-demand, prophylactic, and perioperative treatment of children under 12 years of age. The previous indication was limited to adults and older children more than 12 years of age.

“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. “The newly expanded indication makes IXINITY a viable factor IX option for children living with hemophilia B. 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 new indication is based on a phase 3/4 clinical trial that administered IXINITY as a  prophylactic treatment to previously treated pediatric patients under 12 years of age with severe or moderately severe hemophilia B. Investigators evaluated  pharmacokinetics (PK), safety, and efficacy. According to a company release, the study demonstrated that prophylaxis with IXINITY® was associated with low annualized bleeding rates, effective control of bleeding episodes, consistent PK, and a consistent safety profile.

"IXINITY effectively prevented and controlled bleeding episodes in the Phase 3/4 pediatric study," commented Prof Johnny Mahlangu, BSc, MBBCh, MMed, FCPath, one of the study's principal investigators. "The study supported the efficacy and safety of IXINITY in pediatric patients, and the pharmacokinetics and safety profile were consistent with those observed in adults, although dose adjustment may be needed in pediatric patients. The results thus provide further validation of the clinical utility of IXINITY as a treatment for all people living with hemophilia B."

Learn more about hemophilia B.

Source: Medexus news release dated March 26, 2024