Ixinity (formerly IB1001) is a replacement therapy developed and marketed by Aptevo Therapeutics and approved by the U.S. Food and Drug Administration for the treatment of adults and children 12 years of age or older with hemophilia B.
How Ixinity works
Hemophilia B is a bleeding disorder caused by the deficiency of one of the factors required for blood clotting called factor IX (FIX). This results in delayed blood clotting and, in severe cases, can be life-threatening.
Ixinity is a recombinant human factor IX produced in Chinese hamster ovary (CHO) cells. When injected into hemophilia B patients, Ixinity replenishes FIX levels and restores the normal blood clotting process. It is recommended for the control and prevention of bleeding episodes, and to manage blood loss during surgery in hemophilia B patients.
Ixinity in clinical trials
A multicenter Phase 2/3 clinical trial (NCT00768287) was conducted in 77 hemophilia B patients to determine the pharmacokinetics (movement in the body), safety, and efficacy of Ixinity. Results showed that Ixinity was well-tolerated, the most common adverse event being headache, which was only experienced by 2.6% of participants. There were no reports of FIX inhibitors during the treatment, and Ixinity was effective as a prophylactic treatment. Moreover, one or two infusions of Ixinity resolved 84% of bleeds, and its ability to control these bleeds was rated as excellent or good.
A multicenter, non-randomized, open-label Phase 3 study (NCT01271868) was conducted in hemophilia B patients younger than 12 to evaluate the pharmacokinetics, safety, and efficacy of Ixinity. Results showed that Ixinity was safe and well-tolerated in children. During the trial, 61 bleeding episodes were reported among the 11 patients receiving Ixinity as a prophylactic treatment. Of these, six (10%) resolved with no infusions of Ixinity, 44 (72%) resolved after one infusion of Ixinity, five (8%) required two infusions, and six (10%) required three to five infusions of Ixinity. Bleed control was rated as excellent in 65% of bleeding episodes, good in 32%, and fair in 3%. The most common adverse event was hyperhidrosis (excessive sweating), while one patient experienced fever. Pharmacokinetic analyses showed that Ixinity was metabolized faster and showed lower recovery in patients younger than 12 compared with patients older than 12.
A single-arm, open-label Phase 3/4 trial (NCT03855280) called APVO101-903 is planned to evaluate the pharmacokinetics, safety, and efficacy of Ixinity in preventing or controlling bleeding during prophylaxis in children with severe or moderately severe hemophilia B. For the study, patients will be divided into two groups: those younger than age 6 and those between the ages of 6 and 12. The study is scheduled to begin in August 2019 and completed by August 2021. It has not started recruiting participants yet.
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