Eloctate Performs Well in Phase III Trial of Pediatric Hemophilia A

Long-lasting, investigational hemophilia A therapy Eloctate proved both well-tolerated and efficient at controlling bleeding episodes in a Phase III trial of children under the age of 12, Cambridge, Mass. drugmaker Biogen Idec and partner Swedish Orphan Biovitrum (Sobi) said Thursday.

The open-label, multi-center Kids A-LONG trial evaluated the safety, efficacy and pharmacokinetics of Eloctate (recombinant factor VIII Fc fusion protein) in 71 boys with severe hemophilia A who had at least 50 days of prior exposure to factor VIII treatments at 23 centers across eight countries.

Among the study’s endpoints were the frequency of inhibitor development, projected number of annual bleeding episodes and number of infusions used to prevent and treat bleeds. Children given the drug prophylactically had a median annual projected bleeding rate of 2.0 and a median rate of zero for spontaneous bleeds, with 46 percent of patients experiencing not a single bleeding episode, Biogen says. Additionally, 93 percent of bleeds were controlled with up to two Eloctate infusions.

Kids A-LONG will eventually support regulatory filings for pediatric indications around the world, and particularly in Europe, according to the drugmakers. The drug is also under review in the U.S., Canada, Australia and Japan.

Following regulatory approvals, Biogen Idec will market Eloctate in North America and other regions outside Sobi’s territory, which includes Europe, Russia, the Middle East and North Africa. — Lena Freund

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