The U.S. FDA approved Kovaltryâ„¢ antihemophilic factor (recombinant), an unmodified, full-length factor VIII compound, for the treatment of hemophilia A in children and adults. The drug is infused prophylactically two to three times per week for adolescents and adults and two to three times per week or every other day for pediatric patients to reduce the risk of bleeding.
The approval was based on results from three Long-Term Efficacy Open-Label Program in Severe Hemophilia A Disease (LEOPOLD) clinical trials, which were carried out in more than 200 children and adults with severe hemophilia A from 60 sites in 25 countries worldwide. The drug demonstrated control of and protection from bleeds when used prophylactically two to three times per week in each multi-center, open-label, uncontrolled study. These trials established the pharmacokinetics, safety, and efficacy of the recombinant antihemophilic factor in previously treated children, adolescents, and adults. The most frequently reported adverse events in these trials (reported in â‰¥3% of patients) were headache, pyrexia, and pruritus.
This drug was approved by the European Union earlier this year.
Sources: Bayer press release, March 17, 2016.