It was announced today that ALPROLIX, the first long-acting coagulation factor treatment for hemophilia B, has been approved by Health Canada. ALPROLIX use is for controlling and preventing bleeding occurrences in adults and adolescents.
This is the first worldwide regulatory approval for a long-lasting hemophilia B drug, with approval expected in the U.S. later this year.
Health Canada’s approval was based on the results of the Phase 3 B-LONG study. This study was the largest research on hemophilia B ever done. The study showed that ALPROLIX safely and effectively reduced or prevented bleeding episodes in children over 12 and adults with severe hemophilia B on a treatment regimen of infusions once weekly, or even every 10 to 14 days. A single ALPROLIX infusion controlled over 90 percent of bleeding episodes.
ALPROLIX is the first long-acting fully recombinant clotting factor treatment. The product’s half-life is extended through protein fusion technology, and relies on a natural biological cycling system.
Hemophilia B patients are lacking sufficient quantities of Coagulation Factor IX, which is a natural protein produced by the body that causes blood to clot. Patients with hemophilia A are missing the clotting factor VIII, but the results are the same. Both bleed longer than normal. These prolonged bleeding episodes can cause pain and permanent damage from bleeding into the joints, as well as life-threatening hemorrhages.
Patients with hemophilia B have largely done well with Factor IX injections, but the required frequency of treatment can be a burden that interferes with the ability to lead a normal life. Canada’s approval of the long-acting hemophilia B drug means the reduction by half or even more the number of expensive intravenous infusions now required to control the disease.
George A. Scangos, Ph.D., the chief executive officer of Biogen Idec, said they believe ALPROLIX’s efficacy, safety, and dosing schedule will provide a new way to manage hemophilia.
ALPROLIX will require prophylactic (preventative) infusions as infrequently as once every 10 to 14 days. Current treatment guidelines recommend infusions two or more times per week.
Scangos says ALPROLIX is the first significant hemophilia B treatment advance in 17 years. He said Biogen Idec believe the new drug will give people a meaningful new way to manage their hemophilia B condition.
Not all hemophilia B patients will need the new drug if they are doing well on their current treatment, says Craig M. Kessler, MD, director of the Hemophilia and Thrombophilia Treatment Center at Georgetown University, which was not involved in the study. But some people who are severely affected by hemophilia B may bleed 50 or more times a year, and the new drug could bring this rate close to zero.
Out of 10,000 cases of hemophilia in the U.S., approximately 2,000 are type B. The disease affects an estimated 28,000 people worldwide. It occurs in one out of every 25,000 male births, more rarely in females.
With Canadian approval of the long-acting hemophilia B treatment one big question still to be answered is whether there will truly be a cost to benefit advantage with ALPROLIX. Wide acceptance is probably going to be based largely on price.
By Beth A. Balen