LEVERKUSEN (dpa-AFX) - Bayer HealthCare, a subgroup of Bayer AG (BAYZF.PK, BAYRY.PK, BYR.L) , announced that Bayer Yakuhin, Ltd., Osaka, Japan, has submitted an application for marketing authorization for BAY 81-8973, a recombinant Factor VIII (rFVIII) compound, as a treatment of hemophilia A.
BAY 81-8973 is a full-length recombinant factor VIII which has shown clinical benefit of efficacy when used for prophylaxis twice or three times per week, with standard dosages.
The submission was based on positive results from three Phase III trials in children, adolescents and adults including a total of 204 subjects. The LEOPOLD II clinical trial demonstrated the superiority of prophylaxis versus on-demand therapy with BAY 81-8973. Results show a 93.3 percent reduction in median annualized bleeding rate (ABR) in the twice-per-week prophylaxis arm versus on-demand and a 96.7 percent reduction in the three-times-per-week arm versus on-demand. Patients in this study were randomized between the three treatment arms.
The actual median ABRs observed were four bleeds when treated two times per week and two bleeds when treated three times versus 60 in the on-demand group. Patients treated in both the twice-per-week arm and the three-times-per-week arm maintained adequate bleeding control while using 20 to 40 IU/KG. When used on-demand, 95 percent of bleeds were controlled with one or two infusions. No clinically relevant treatment-related adverse events occurred and no inhibitor formation was observed.
In LEOPOLD Kids (pediatric trial) including 51 pre-treated children <12 years of age, BAY 81-8973 demonstrated good efficacy with twice or three times per week or every other day prophylaxis regimens, and no inhibitors were observed. A clinical study in previously untreated patients is ongoing.
Hemophilia A, also known as factor VIII deficiency or classic hemophilia is a largely inherited bleeding disorder. Hemophilia A, the most common type of hemophilia, is caused by deficient or defective blood coagulation proteins, known as Factor VIII. Hemophilia A is characterized by prolonged or spontaneous bleeding, especially into the muscles, joints or internal organs.
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