Baxter International has filed an application to the US Food and Drug Administration (FDA) for a paediatric indication for Rixubis, Coagulation Factor IX (Recombinant), for the treatment of hemophilia B.
The filing was based on a Phase II/III clinical trial evaluating the efficacy and safety of Rixubis in 23 previously-treated male patients less than 12 years of age with severe or moderately severe hemophilia B.
Earlier this year, Rixubis was approved in the US for adults with hemophilia B earlier and the company filed for marketing approval in Europe in this November.
Pomeranian Medical University’s Department of Paediatrics, Haematology and Oncology lead investigator Tomasz Urasinski said routine prophylactic infusions can be particularly beneficial for children with hemophilia, particularly if they are started early to help prevent joint bleeding.
"The clinical evidence suggests that Rixubis prophylaxis can offer hemophilia B patients a reduction in bleeding episodes while also being tolerable, an important factor for this young population," Urasinski said.
In the trial, patients were treated with a twice-weekly Rixubis prophylaxis regimen (median dose 56 IU/kg) over six months or for a minimum of 50 exposure days (EDs).
The company said that the median annualised bleeding rate (ABR) was 2.0 (0.0 for spontaneous bleeds and joint bleeds).
In the trial, nine patients (39.1%) experienced no bleeding and 23 patients (88.5%) were treated with one to two infusions.
According to the company, out of the 26 bleeds seen in the trial, only two (in two patients) were spontaneous and no reports of inhibitor development, no allergic reactions, and no thrombotic or treatment-related adverse events were observed among the study participants.
Baxter BioScience vice-president of global R&D Anders Ullman said the positive results among a pediatric patient population are consistent with those observed in the Rixubis pivotal study among adult patients with hemophilia B.
"We submitted this data as part of our application for a pediatric indication for Rixubis to advance effective therapeutic solutions for children with hemophilia B," Ullman said.
Rixubis is a recombinant factor IX (rFIX) protein for both routine prophylaxis and control of bleeding episodes in adults with hemophilia B and it is the first new rFIX approved to treat hemophilia B in more than 15 years as well as the first rFIX approved by the FDA for both routine prophylaxis and control of bleeding episodes in the chronic condition.