The FDA has granted approval for the use of hemophilia B treatment Rixubis in pediatric patients.
The FDA has granted approval for the use of hemophilia B treatment Rixubis [coagulation factor IX (recombinant)] in pediatric patients.
The treatment, which was previously approved in June 2013 for use in patients aged 16 years and older, now offers younger patients routine prophylactic treatment, control, and prevention for their bleeding episodes.
''In addition to the positive reception we've received from adult Rixubis patients, the approval for pediatric patients offers a valuable new option, particularly as our clinical data demonstrated a significant reduction in bleeding episodes for patients who were treated prophylactically, an important factor for this young patient population,'' said John Orloff, MD, vice president of global research and development at Baxter BioScience, in a press release.
Hemophilia B, which affects approximately 3300 people in the United States, is caused by mutations in the factor IX gene and leads to deficiency of factor IX. Rixubis, a purified protein produced by recombinant DNA technology, is supplied in single-use vials administered by intravenous injection after reconstitution with sterile water for injection.
Rixubis’s expanded approved was based on a clinical trial that established its efficacy and safety in 23 previously treated male patients under 12 years of age with severe or moderately severe hemophilia B. The study participants received a twice-weekly Rixubis prophylaxis regimen (mean dose 56 IU/kg) over a mean treatment period of 6 months and a mean 54 exposure days.
The study showed the median annualized bleeding rate was 2.0, with a rate of 0.0 for spontaneous bleeds and joint bleeds. During the study, 9 patients experienced no bleeds, while 23 bleeding episodes were treated with 1-2 infusions.
There were no incidents of inhibitor development, severe allergic reactions, or thrombotic or treatment-related adverse events among the participants.
Approximately 1% of subjects experienced dysgeusia, extremity pain, and a positive test for furin antibody.