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news.CSL Behring has submitted a biologics license application (BLA) to the US Food and Drug Administration (FDA) seeking marketing authorization of its long-acting fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP).
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Following approval, rIX-FP will provide people with hemophilia B and their physicians a long-acting treatment option with dosing intervals up to 14 days.
The BLA is based on the results from the open-label, multicenter, safety, pharmacokinetic (PK) and efficacy PROLONG-9FP Phase II/III trial of rIX-FP in previously treated patients (aged 12 to 61 years) with severe hemophilia B.
CSL chief scientific officer and R&D director Dr Andrew Cuthbertson said: "As we mentioned at our recent R&D investor briefing, submission of our BLA to the FDA for rIX-FP is a significant milestone for CSL Behring’s recombinant factor IX development program and moves us one step closer to bringing this innovative therapy to hemophilia B patients in the US.
"Our strong partnership with and commitment from the hemophilia community led us to develop rIX-FP based on novel recombinant albumin fusion technology.
"This technology has led to a long-acting treatment candidate that continues our legacy of improving the well-being of patients with bleeding disorders and other rare diseases."
The Phase II/III trial was designed to compare the change in frequency of spontaneous bleeding events between on-demand treatment and a weekly prophylaxis regimen in patients previously receiving only on-demand treatment
The trial, which evaluated multiple prophylaxis regimens including 7-day and 14-day intervals, compared the number of patients developing inhibitors against factor IX as primary outcome measures.