GTC Biotherapeutics (fka GTCB)

[Lewis R Goudy]

NovoSeven item

Decreased Bleeding Frequency With Secondary Prophylactic Use Of NovoSeven
http://www.medicalnewstoday.com/medicalnews.php?newsid=58798&nfid=nl

Data presented at the 48th Annual Meeting of the American Society of Hematology (ASH) from a study testing the use of NovoSeven® Coagulation Factor VIIa (Recombinant) for secondary prophylaxis in patients with hemophilia complicated by inhibitors. The study showed that once-daily NovoSeven®, when used as secondary prophylaxis for a three-month period in patients with hemophilia A or B complicated by inhibitors, significantly reduced the frequency of bleeding episodes, particularly spontaneous joint bleeds, compared with acute treatment only when a bleed occurred. Secondary prophylaxis is when a treatment is used for a defined period of time in anticipation of reducing repeated bleeding, unlike primary prophylaxis, which is when a treatment is used to reduce or prevent joint disease.

"Prophylactic use of clotting factors in hemophilia patients without inhibitors is a widely accepted treatment. For hemophilia patients with inhibitors, treatment is still usually confined to on-demand therapy to treat bleeds," said Ulla Hedner MD, of the University of Lund, Sweden, and vice president of Research & Development of Novo Nordisk A/S, Denmark. "These results are encouraging and indicate there may be potential benefits offered by prophylactic treatment with NovoSeven® to hemophilia patients with inhibitors."

Study and Findings

Bleed frequency was reduced by 45% and 59% with a daily dose of NovoSeven® at either 90ug/kg or 270�g/kg respectively (p<0.0001 for both), compared with the pre-treatment three-month observation period during which therapy was only used at the time of a bleed. In addition, clinically relevant reductions in bleeding were also maintained during the three-month observation period immediately following the three-month treatment period during which only on-demand therapy was used. Bleed frequency was reduced by 27% at 90ug/kg (p<0.0001) and by 50% at 270ug/kg (p<0.0001).1

Data were obtained from a prospective, randomized, double-blind, uncontrolled trial, which studied 22 patients who experience at least four bleeds per month. These results are from the first-ever prospective trial designed to investigate the potential benefits of secondary prophylaxis with a bypassing agent in patients with hemophilia A or B complicated by inhibitors.

Hemophilia patients who have inhibitors (or neutralizing antibodies) against clotting-replacement factor VIII or factor IX experience severe morbidity due to their frequent joint, muscle and deep tissue bleeds and subsequently impaired joint function. They also have frequent pain, disability, regular hospitalizations and considerable absence from school or work.

"Some hemophilia patients with inhibitors, like hemophilia patients without inhibitors, experience frequent bleeds," continued Hedner. "In this study daily use of NovoSeven® for a period of three months offered improvements in bleed frequency."

Secondary prophylaxis with NovoSeven® was not associated with any unexpected safety concerns, and no thromboembolic events were reported during the study.
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