Ixinity up for FDA approval for children under 12 with hemophilia B
Therapy is already OK'd as prophylactic, on-demand treatment for adults, teens
Ixinity is already approved by the FDA for adults and adolescents 12 and older. The therapy is indicated for use as a routine prophylactic, or preventive treatment, to reduce the frequency of bleeds, as well as an on-demand treatment to treat and control bleeds during surgery. The new application seeks to extend its approval in these indications for younger patients.
“Approximately one third of patients treated for hemophilia B in the United States are children 13 years of age and under. Risk of bleeding events can severely limit these children’s daily activities,” Ken d’Entremont, CEO of Medexus, said in a company press release. “The FDA’s commitment to review our [supplemental biologics license application] brings us a step closer to making Ixinity a viable factor IX treatment option for this important population.”
Medexus didn’t indicate when a decision can be expected. It typically takes about 10 months for an application to be reviewed.
Ixinity a lab-made FIX
Hemophilia B is caused by mutations in the gene that provides instructions for making the factor IX (FIX) blood clotting protein. Without it blood is unable to clot properly, leading to an increased risk of excessive and prolonged bleeding.
Ixinity is a replacement therapy that contains a recombinant, or lab-made, version of FIX that can be given to help prevent or control bleeds with hemophilia B. It’s administered via an infusion into the bloodstream, with various dosing protocols that depend on the specific situation.
In its announcement that the FDA had agreed to review its request, Medexus didn’t state what data formed the basis of the application. The company has conducted a Phase 4 trial (NCT03855280) that tested Ixinity in more than 20 young children with hemophilia B. Data from other studies have suggested Ixinity’s safety and effectiveness in younger children is comparable to older patients.