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FDA partially rescinds two approvals after orphan exclusivity mix-up

Posted 24 February 2021 | By Michael Mezher 

FDA partially rescinds two approvals after orphan exclusivity mix-up

The US Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER) last week partially rescinded the approval of two supplemental biologics license applications (sBLAs) for recombinant factor IX products after determining that part of the products’ indications were covered by orphan drug exclusivity.
Last year, FDA approved sBLAs for Wyeth Pharmaceuticals’ BeneFIX and Aptevo BioTherapeutics’ Ixinity, granting both products new indications for routine prophylaxis in adults and children with hemophilia B to reduce the frequency of bleeding episodes.
However, in letters to both companies dated 17 February, FDA explains that it erred in approving the pediatric portion of the prophylaxis indications because they were covered under the seven years of orphan drug exclusivity awarded to Baxalta’s recombinant factor IX product Rixubis.
In both letters, FDA notes that the adult prophylaxis indication approved under the sBLAs “remains in effect” and that the orphan drug exclusivity for Rixubis will be in effect until 12 September 2021.
FDA first approved Rixubis as the first recombinant factor IX treatment for routine prophylaxis of hemophilia B in 2013, and in 2014 expanded its use to children. Rixubis was granted orphan drug designation for prophylactic use to prevent or reduce the frequency of bleeding episodes in patients with hemophilia B for both its initial approval and subsequent expansion. The orphan drug exclusivity for the adult indication expired on 26 June 2020.
“Upon review of Agency records, we have determined that FDA erred in approving the indication for routine prophylaxis in children with hemophilia B (congenital factor IX deficiency or Christmas disease) on June 26, 2020,” FDA wrote to Wyeth Pharmaceuticals. The agency made a similar statement in its letter to Aptevo regarding its sBLA approved on 25 September 2020.
FDA says the letters have been sent to correct its error and clarify that the sBLA approvals only cover the adult prophylaxis indication and to request that the companies revise their labeling to remove the portion of the indication protected by orphan drug exclusivity. FDA asks both companies to submit updated labeling as part of a Supplement – Changes Being Effected amendment including all revisions since the last approval of prescribing information.
Wyeth, Aptevo


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