PHARMACY

Biogen Idec drugs could advance treatment of hemophilia, clinical trials find

BY Alaric DeArment

NEW YORK — Two experimental drugs under development by Biogen Idec are effective in reducing bleeding episodes in patients with two forms of hemophilia when administered as a preventive treatment, while also reducing the need for frequent injections, according to late-stage clinical trial data.

The two phase-3 studies were published in the November issue of the journal Blood and the December issue of the New England Journal of Medicine. The Blood trial, titled "A-LONG," tested Biogen Idec’s recombinant factor VIII Fc fusion protein in patients with hemophilia A, while the NEJM trial, titled "B-LONG", tested recombinant factor IX Fc fusion protein in patients with hemophilia B.

"Results from these largest global phase-3 pivotal trials demonstrate the potential of revolutionizing the hemophilia treatment paradigm by offering longer protection from bleeding episodes while reducing the frequency of intravenous injections," clinical lead Alvin Luk told DSN.

The hemophilia A trial divided 165 patients into three groups, one of which received a dose of the drug every 3-5 days per week on a preventive basis, known as prophylaxis; another prophylaxis group that received a dose every week; and another that received the drug on an as-needed basis as bleeding episodes were occurring. Any patients in any group could use the drug for major surgeries. Patients who received prophylaxis had significantly reduced bleeding rates compared with those who received it on an episodic basis.

The 123 hemophilia B patients were divided into a group that received the weekly prophylaxis; a second group that received interval-adjusted prophylaxis every 10 days or longer; a third group that received treatment as needed; and a fourth that received it for surgical procedures. In the first, second and third groups, 90.4% of bleeding episodes were resolved after one injection, and the responses were good or excellent among patients undergoing surgery.

 

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Novo Nordisk drug for rare hemophilia gets FDA nod

BY Alaric DeArment

SILVER SPRING, Md. — The Food and Drug Administration has approved a new treatment made by Danish drug maker Novo Nordiskfor a rare, genetic bleeding disorder, the agency said Monday.

The FDA announced the approval of Tretten (coagulation factor XIII A-subunit [recombinant]), calling it the first recombinant product for routine prevention of bleeding in adults and children with congenital factor XIII A-subunit deficiency. Factor XIII is a protein involved in blood clotting, and patients with the condition don’t make enough of it. The protein comprises two subunits, A and B; most cases of factor XIII deficiency result from a deficiency of the A-subunit. According to the National Hemophilia Foundation, factor XIII deficiency may be the rarest of all factor deficiencies, affecting one-in-5 million people.

"The approval of this product provides another therapeutic option for the prevention of bleeding in patients with factor XIII A-subunit deficiency," FDA Center for Biologics Evaluation and Research director Karen Midthun said. "Without treatment, people with this rare condition are at risk for serious and life-threatening bleeding."

 

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FDA move allows Ariad Pharmaceuticals’ resumed marketing of Iclusig

BY Alaric DeArment

CAMBRIDGE, Mass. — The Food and Drug Administration has approved revisions to the labeling of a leukemia drug made by Ariad Pharmaceuticals that allow the company to resume selling it, the company said.

Ariad said the FDA approved revised prescribing information and a risk evaluation and mitigation strategy, or REMS, for Iclusig (ponatinib) in refractory Philadelphia chromosome-positive, or Ph+ leukemias. These conditions include T315I-positive chronic myeloid leukemia; T315I-positive Ph+ acute lymphoblastic leukemia; and chronic-phase, accelerated-phase or blast-phase chronic myeloid leukemia; or Ph+ acute lymphoblastic leukemia in patients for whom no other similar therapy can be used.

"In less than two months of suspending marketing and commercial distribution of Iclusig in the [United States], we addressed the issues raised by the FDA and now are able to market and distribute Iclusig again in the [United States]," Ariad chairman and CEO Harvey Berger said. "As we look ahead to relaunching Iclusig in the [United States] and fulfilling our post-marketing requirements, we will continue to focus on understanding the benefits and risks of Iclusig treatment in patients with resistant or intolerant Philadelphia-positive leukemias."

 

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