The Food and Drug Administration (FDA) has approved revised labeling for BeneFIX (coagulation factor IX [recombinant]) that removes the indication for routine prophylaxis to reduce the frequency of bleeding episodes in children with hemophilia B (congenital factor IX deficiency or Christmas disease) less than 16 years of age.

Following a review of records, the FDA partially rescinded the approval of BeneFIX for routine prophylaxis with hemophilia B in February 2021 indicating that the product was not eligible to be approved for this indication in children at that time. The Agency stated that it “erred in approving the indication for routine prophylaxis in children with hemophilia B” due to the Orphan Drug exclusivity that was granted to Rixubis (coagulation factor IX [recombinant]; Baxalta US Inc).

According to the letter, the indication for routine prophylaxis in adults 16 years of age and older with hemophilia B is “not at issue”, and “remains in effect.” BeneFIX is also indicated for adults and children with hemophilia B for on-demand treatment and control of bleeding episodes and perioperative management of bleeding.

The FDA’s Orphan Drug provisions block the approval of the same product for the same indication for 7 years. The exclusivity for Rixubis remains in effect until September 12, 2021.


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References

  1. US Food and Drug Administration, Center for Biologics Evaluation and Research. BL 103677/6937 supplement approval letter. April 20, 2021. https://www.fda.gov/media/147917/download
  2. US Food and Drug Administration, Center for Biologics Evaluation and Research. BL 103677/6937 partial rescission of approval letter. February 17, 2021. https://www.fda.gov/media/146120/download.