Biogen Idec and Swedish Orphan Biovitrum AB (Sobi) recently announced positive results from its late-stage hemophilia B study.
The companies announced top-line results of the Kids B-LONG Phase III clinical study that evaluated the safety, efficacy and pharmacokinetics of Alprolix [Coagulation Factor IX (Recombinant), Fc Fusion Protein] in children under the age of 12 with severe hemophilia B. According to the companies, the successful completion of the late-stage study supports applications for pediatric indications in several locations.
“According to published studies, prophylactic treatment for children with severe hemophilia is recommended because it is associated with proven clinical benefits. However, frequent administration schedules can be burdensome for children and their caregivers,” said Aoife Brennan, MD, vice president of hematology clinical development at Biogen Idec. “These data will enable regulatory filings in Europe later this year as well as support pediatric indications in other countries, with the potential to help address a critical need among children with hemophilia B.”
The drug was generally well tolerated and no inhibitors were detected during the study, meeting the primary endpoint of the study. In the Kids B-LONG trial, children treated prophylactically with Alprolix demonstrated an overall median annualized bleeding rates (ABR) of 1.97. The median ABR for spontaneous joint bleeds was zero. Approximately 33 percent of participants in the study experienced zero bleeding episodes. Overall, 91.7 percent of bleeding episodes were controlled by one or two infusions of Alprolix. The terminal half-life of the drug in the study was 66.5 hours for children under six and 70.3 hours for children six to less than 12 years of age.
Additional analyses of the study are still ongoing. The companies said that detailed results will be presented at a future scientific meeting. Interim results from the study helped support the US approval of Alprolix for use in children.
“Sobi and Biogen Idec are committed to advancing treatment options for adults and children with haemophilia,” said Birgitte Volck, MD, PhD, senior vice president of development and chief medical officer at Sobi. “The completion of Kids B-LONG marks an important milestone in the Alprolix development program. We are excited to pursue the next stages of preparation for regulatory filing in Europe and to potentially advance treatment options for people with haemophilia B.”
Hemophilia B occurs in about one in 25,000 male births annually, and more rarely in females, affecting about 6,300 people in the US. Approximately 28,000 people are currently diagnosed with hemophilia B according to estimates from the World Federation of Hemophilia. The disease is caused by having substantially reduced or no factor IX activity, which is needed for normal blood clotting. Hemophilia B patients experience bleeding episodes that cause pain, irreversible joint damage and life-threatening hemorrhages.
Source: Biogen Idec
Last updated: 3/2/15; 10:45am EST