FDA Approves Medexus’s Supplemental Biologics License Application for IXINITY(R) to Treat Hemophilia B in Pediatric Patients

Toronto, Ontario and Chicago, Illinois — Medexus Pharmaceuticals (TSX: MDP) (OTCQX: MEDXF) today announced that the US Food and Drug Administration (FDA) recently approved Medexus’s supplemental Biologics License Application (sBLA) for IXINITY® [coagulation factor IX (recombinant)] for the on-demand, prophylactic, and perioperative treatment of pediatric patients under 12 years of age with hemophilia B. IXINITY®, an intravenous recombinant factor IX therapeutic, is now approved for use in all patients with hemophilia B, a hereditary bleeding disorder characterized by a deficiency of clotting factor IX in the blood.

“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 IXNITY 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.”

“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.”

The sBLA and expanded indication for IXINITY® are based on results from a Phase 3/4 study that evaluated the pharmacokinetics (PK), safety, and efficacy of IXINITY® as a prophylactic treatment in previously treated pediatric patients under 12 years of age with severe or moderately severe hemophilia B. 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. For more information about the study’s methods, results, and conclusions, see “About the study” in Medexus’s August 17, 2023 press release, available on the Investors-News & Events section of Medexus’s corporate website.



IXINITY® [coagulation factor IX (recombinant)] Lyophilized Powder for Solution for Intravenous Injection is a coagulation factor IX (recombinant) indicated in adults and children with hemophilia B for on-demand treatment and control of bleeding episodes, perioperative management, and for routine prophylaxis to reduce the frequency of bleeding episodes.

The most common adverse reaction observed in >2% of patients in clinical trials was headache. For more information about IXINITY®, including important safety information, see the full prescribing information, which is available on the product’s website at: www.ixinity.com.

IXINITY® is approved by the FDA for sale and use in the United States only and is not intended for export outside the United States. Medexus makes no representation that IXINITY® is appropriate for, or authorized for sale to or use by, persons who are not located in the United States.


About hemophilia B

Hemophilia B is a genetic bleeding disorder caused by a deficiency of coagulation factor IX, a protein needed to produce blood clots to stop bleeding. The clinical spectrum may include spontaneous or trauma-induced bleeding into joints, muscles, and soft tissues, resulting in joint damage, reduction in mobility, and severe arthritis, all of which negatively impact health-related quality of life. The primary aim of care is to prevent and treat bleeding by replacing the deficient clotting factor.


About Medexus

Medexus is a leading specialty pharmaceutical company with a strong North American commercial platform and a growing portfolio of innovative and rare disease treatment solutions. Medexus’s current focus is on the therapeutic areas of oncology, hematology, rheumatology, auto-immune diseases, allergy, and dermatology.



Ken d’Entremont | CEO, Medexus Pharmaceuticals
Tel: 905-676-0003 | Email: [email protected]

Marcel Konrad | CFO, Medexus Pharmaceuticals
Tel: 312-548-3139 | Email: [email protected]

Victoria Rutherford | Adelaide Capital
Tel: 480-625-5772 | Email: [email protected]