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The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended BAY 81-8973, a recombinant factor VIII (FVIII) compound, for approval in the European Union.
BAY 81-8973 is an unmodified, full-length, recombinant FVIII compound intended for the treatment and prophylaxis of bleeding in hemophilia A patients of all ages.
The CHMP’s recommendation has been relayed to the European Commission, which is expected to make a decision regarding BAY 81-8973 in the coming weeks.
If approved, BAY 81-8973 will be marketed for the aforementioned indication in the 28 member countries of the European Union, as well as Iceland, Liechtenstein, and Norway.
The CHMP’s recommendation is based on results from the LEOPOLD trials, which were presented at the National Hemophilia Foundation’s 67th Annual Meeting last August.
LEOPOLD trials
The LEOPOLD Clinical Development Program consists of 3 multinational clinical trials designed to evaluate the pharmacokinetics, efficacy, and safety of BAY 81-8973 in subjects with severe hemophilia A (<1% FVIII:C).
LEOPOLD I is an open-label, cross-over, phase 3 study of males ages 12 to 65. The objectives were to demonstrate the efficacy and safety of BAY 81-8973 when used as prophylaxis, for the treatment of bleeding episodes, and for maintaining hemostasis during surgery. In LEOPOLD I, investigators assigned subjects to either the 2- or 3-times-weekly dosing regimens based on each patient’s phenotype, prior bleeding history and other factors.
LEOPOLD II is a randomized, cross-over, open-label trial conducted in male subjects ages 12 to 65. In this phase 3 study, 80 subjects were randomized to receive BAY 81-8973 either as a low-dose prophylaxis regimen (20-30 IU/kg; n=28) twice per week, high-dose prophylaxis (30-40 IU/kg; n=31) 3 times a week, or on-demand (n=21).
The primary objective was to demonstrate the superiority of prophylaxis over on-demand therapy, with the primary endpoint being bleeding frequency at 12 months.
LEOPOLD Kids is an open-label, non-randomized, phase 3 study. Part A is designed to evaluate the efficacy and safety of BAY 81-8973 for prophylaxis, treatment of bleeds, and surgical management in previously treated children at least 12 years of age with twice or 3 times per week or every other day prophylaxis regimens. Part B of the study, which involves previously untreated patients, is ongoing.
Bayer HealthCare AG has submitted marketing applications for BAY 81-8973 in the US and several other countries and is pursuing regulatory approvals worldwide. 
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended BAY 81-8973, a recombinant factor VIII (FVIII) compound, for approval in the European Union.
BAY 81-8973 is an unmodified, full-length, recombinant FVIII compound intended for the treatment and prophylaxis of bleeding in hemophilia A patients of all ages.
The CHMP’s recommendation has been relayed to the European Commission, which is expected to make a decision regarding BAY 81-8973 in the coming weeks.
If approved, BAY 81-8973 will be marketed for the aforementioned indication in the 28 member countries of the European Union, as well as Iceland, Liechtenstein, and Norway.
The CHMP’s recommendation is based on results from the LEOPOLD trials, which were presented at the National Hemophilia Foundation’s 67th Annual Meeting last August.
LEOPOLD trials
The LEOPOLD Clinical Development Program consists of 3 multinational clinical trials designed to evaluate the pharmacokinetics, efficacy, and safety of BAY 81-8973 in subjects with severe hemophilia A (<1% FVIII:C).
LEOPOLD I is an open-label, cross-over, phase 3 study of males ages 12 to 65. The objectives were to demonstrate the efficacy and safety of BAY 81-8973 when used as prophylaxis, for the treatment of bleeding episodes, and for maintaining hemostasis during surgery. In LEOPOLD I, investigators assigned subjects to either the 2- or 3-times-weekly dosing regimens based on each patient’s phenotype, prior bleeding history and other factors.
LEOPOLD II is a randomized, cross-over, open-label trial conducted in male subjects ages 12 to 65. In this phase 3 study, 80 subjects were randomized to receive BAY 81-8973 either as a low-dose prophylaxis regimen (20-30 IU/kg; n=28) twice per week, high-dose prophylaxis (30-40 IU/kg; n=31) 3 times a week, or on-demand (n=21).
The primary objective was to demonstrate the superiority of prophylaxis over on-demand therapy, with the primary endpoint being bleeding frequency at 12 months.
LEOPOLD Kids is an open-label, non-randomized, phase 3 study. Part A is designed to evaluate the efficacy and safety of BAY 81-8973 for prophylaxis, treatment of bleeds, and surgical management in previously treated children at least 12 years of age with twice or 3 times per week or every other day prophylaxis regimens. Part B of the study, which involves previously untreated patients, is ongoing.
Bayer HealthCare AG has submitted marketing applications for BAY 81-8973 in the US and several other countries and is pursuing regulatory approvals worldwide.
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended BAY 81-8973, a recombinant factor VIII (FVIII) compound, for approval in the European Union.
BAY 81-8973 is an unmodified, full-length, recombinant FVIII compound intended for the treatment and prophylaxis of bleeding in hemophilia A patients of all ages.
The CHMP’s recommendation has been relayed to the European Commission, which is expected to make a decision regarding BAY 81-8973 in the coming weeks.
If approved, BAY 81-8973 will be marketed for the aforementioned indication in the 28 member countries of the European Union, as well as Iceland, Liechtenstein, and Norway.
The CHMP’s recommendation is based on results from the LEOPOLD trials, which were presented at the National Hemophilia Foundation’s 67th Annual Meeting last August.
LEOPOLD trials
The LEOPOLD Clinical Development Program consists of 3 multinational clinical trials designed to evaluate the pharmacokinetics, efficacy, and safety of BAY 81-8973 in subjects with severe hemophilia A (<1% FVIII:C).
LEOPOLD I is an open-label, cross-over, phase 3 study of males ages 12 to 65. The objectives were to demonstrate the efficacy and safety of BAY 81-8973 when used as prophylaxis, for the treatment of bleeding episodes, and for maintaining hemostasis during surgery. In LEOPOLD I, investigators assigned subjects to either the 2- or 3-times-weekly dosing regimens based on each patient’s phenotype, prior bleeding history and other factors.
LEOPOLD II is a randomized, cross-over, open-label trial conducted in male subjects ages 12 to 65. In this phase 3 study, 80 subjects were randomized to receive BAY 81-8973 either as a low-dose prophylaxis regimen (20-30 IU/kg; n=28) twice per week, high-dose prophylaxis (30-40 IU/kg; n=31) 3 times a week, or on-demand (n=21).
The primary objective was to demonstrate the superiority of prophylaxis over on-demand therapy, with the primary endpoint being bleeding frequency at 12 months.
LEOPOLD Kids is an open-label, non-randomized, phase 3 study. Part A is designed to evaluate the efficacy and safety of BAY 81-8973 for prophylaxis, treatment of bleeds, and surgical management in previously treated children at least 12 years of age with twice or 3 times per week or every other day prophylaxis regimens. Part B of the study, which involves previously untreated patients, is ongoing.
Bayer HealthCare AG has submitted marketing applications for BAY 81-8973 in the US and several other countries and is pursuing regulatory approvals worldwide.