Biogen Idec and Swedish Orphan Biovitrum plan to advance the companies' long-lasting, fully-recombinant factor VIII Fc fusion protein (rFVIIIFc) into a registrational clinical trial in people with haemophilia A. The decision to advance the programme is based on promising data from a phase-1/2a open-label, cross-over, multi-center, dose-escalation study that evaluated the safety and pharmacokinetics of an intravenous injection of rFVIIIFc in 16 previously-treated patients with severe haemophilia A. In the study, rFVIIIFc was well tolerated and demonstrated a prolonged half-life compared to Advate (antihemophilic factor recombinant, plasma/albumin-free method, rFVIII), supporting advancement of the program.

The primary objective of the phase-1/2a study was to assess the safety of rFVIIIFc at different doses; the secondary objective was to estimate the pharmacokinetic parameters of rFVIIIFc at doses ranging from 25 to 65 IU/kg. Preliminary results demonstrated that rFVIIIFc's prolonged half-life compared to Advate was seen consistently across all patients and dose levels, and other measures of pharmacokinetics including mean residence time and area under the curve were also increased. There were no signs of injection site reactions, inhibitor development or anti-rFVIIIFc drug antibodies in the single-dose study, and there have been no reports of drug-related serious adverse events.

The treatment of severe haemophilia A requires frequent injections, creating a significant burden for individuals with the disorder. The potential of rFVIIIFc, which is based on Biogen Idec's novel and proprietary monomeric Fc-fusion technology, to prolong protection from bleeding and to reduce the frequency of injections needed for treatment will be evaluated in the registrational trial. The global trial, which will commence following communications with regulatory authorities, is being designed to assess the safety, pharmacokinetics and efficacy of rFVIIIFc in both the prevention and treatment of bleeding in haemophilia A patients.

"Biogen Idec is committed to transforming the lives of people with hemophilia by developing a portfolio of long-lasting therapies," said Glenn Pierce, vice president and chief medical officer of Biogen Idec's hemophilia therapeutic area. "We are excited to announce the advancement of rFVIIIFc into a registrational study. Like our long-lasting hemophilia B program, which entered a registrational trial earlier this year, rFVIIIFc offers the potential to make a significant difference in the lives of people with hemophilia."

"The phase-1/2a results are very encouraging, and the decision to initiate our second registrational program in hemophilia represents strong progress in our efforts to improve the lives of individuals with hemophilia," said Peter Edman, chief scientific officer of Swedish Orphan Biovitrum. "This is also an important milestone in the ongoing development of Swedish Orphan Biovitrum."

In June, the European Medicines Agency's Committee for Orphan Medicinal Products issued a positive opinion regarding the orphan drug application for rFVIIIFc. A final decision is expected in the coming weeks.

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Swedish Orphan Biovitrum is a Swedish-based niche specialty pharmaceutical company with an international market presence.