Late Friday afternoon, Biogen Idec announced
the FDA has granted orphan drug approval of Eloctate [Antihemophilic Factor (Recombinant), Fc Fusion Protein] for the control and prevention of bleeding episodes, perioperative (surgical) management and routine prophylaxis in adults and children with hemophilia A.
Hemophilia A is a rare, chronic, genetic disorder that results in impaired clotting mechanisms due to missing or reduced levels of factor VIII. People with hemophilia A experience recurrent and extended bleeding episodes that cause pain and irreversible joint damage. Some of these bleeding episodes can be life-threatening. There are an estimated 16,000 people in the United States with hemophilia A (and 142,000 world wide). Orphan approved drugs are available to treat bleeding episodes and two orphan drugs - Advate
- are approved for prophylactic use in patients with hemophilia A.
The recommended starting prophylactic regimen for Eloctate is 50 IU/kg every 4 days. Based on clinical response, the dose may be adjusted in the range of 25 to 65 IU/kg, every 3 to 5 days.
In a press release
, Patrick F. Fogarty, MD, assistant professor of medicine at the Hospital of the University of Pennsylvania, and director, Penn Comprehensive Hemophilia and Thrombosis Program, said, “Prophylactic treatment is recommended for people with severe hemophilia, and following a protective regimen can be burdensome given the frequency of infusions required,” adding “Infusion frequency is a major challenge for people with hemophilia, and I believe ELOCTATE begins to address this burden while protecting against bleeding episodes.”
Since Eloctate can be administered twice weekly instead of current treatment options that require dosing every other day, Eloctate may be the preferred prophylactic option for some patients.
“We are encouraged by the arrival of new and innovative therapies, which may help address treatment gaps for the approximately 16,000 adults and children living with hemophilia A in the United States,” said Val Bias, chief executive officer of the National Hemophilia Foundation (NHF). “These advances, in conjunction with continued community education and empowerment, are critical to effectively serving the needs of people with the disorder.”
The approval of ELOCTATE is based on results from the global, Phase 3 A-LONG clinical study, as well as interim analysis from the Phase 3 Kids A-LONG study.
A-LONG and Kids A-LONG Studies
The pivotal trial that led to drug approval was the A-LONG study, which was an open-label, multi-center study that examined the efficacy, safety and PK of Eloctate in 165 previously treated males 12 years of age and older with severe hemophilia A. Results showed that adults and adolescents with severe hemophilia A achieved a statistically significant reduction of bleeding episodes in both of the study’s prophylaxis arms, relative to the on-demand treatment arm. In addition, 98 percent of bleeding episodes were controlled with one or two Eloctate infusions.
The pediatric indication was based on interim safety and PK results in 38 boys ages 2 to 11 years old from the Phase 3 Kids A-LONG study. These data showed that Eloctate was generally well-tolerated and no inhibitors were detected. Children 2 to 5 years old have a shorter half-life and higher clearance of hemophilic factors (adjusted for body weight); therefore, a higher dose or more frequent dosing may be needed in this age group. Since the FDA review, addition data has become available
, showing the drug to be safe and effective in children.
Biogen Idec plans to make Elocate commercially available to people with hemophilia A in the United States in July 2014. Biogen Idec also plans to have a ‘MyEloctate’ service available to help patients.