FDA Expands Indication for Octapharma’s WILATE to Hemophilia A

The U.S. Food and Drug Administration (FDA) has approved WILATE for treatment of adults and adolescents with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes and on-demand treatment and control of bleeding episodes. WILATE was previously approved by FDA in 2009 for treatment of children and adults with von Willebrand disease for on-demand treatment and control of bleeding episodes, as well as for perioperative management of bleeding.

This expanded indication of WILATE is based on results of the Clinical Study to Investigate the PK, Efficacy and Safety of WILATE in Patients with Severe Hemophilia. In the study, 136 previously treated patients with hemophilia A (aged 11 years to 66 years) received WILATE in five clinical studies that involved prophylactic use, treatment on demand, surgery and/or pharmacokinetics. All subjects were male. Overall, subjects received 19,317,004 international units (IU) of WILATE during 9,001 exposure days. The most common adverse reaction was pyrexia (two subjects; 1.5 percent). Further adverse reactions included pruritus, headache and sleeping disorder (one subject; 0.75 percent). Two out of 55 subjects (3.6 percent) in the pivotal study of routine prophylaxis in severe hemophilia A had unexplained transient worsening of pre-existing thrombocytosis while on the study.

“Octapharma has been committed to providing U.S. hemophilia A patients with complete access to the company product portfolio since our inception,” said Octapharma USA President Flemming Nielsen. “We are excited for providers and patients who have been looking forward to the day when WILATE would be indicated for hemophilia A. Octapharma is dedicated to providing the bleeding disorders community with the therapies and programs that enhance patient lives every day.”