Biogen Idec has received approval from the US Food and Drug Administration (FDA) for its long-acting haemophilia B treatment Alprolix [Coagulation Factor IX (Recombinant), Fc Fusion Protein].
The drug is for the control and prevention of bleeding episodes, perioperative (surgical) management and routine prophylaxis in adults and children with haemophilia B.
According to Biogen Idec, the DNA derived haemophilia B therapy is clinically proven to reduce bleeding episodes with prophylactic (protective) infusions starting at least a week apart.
Developed using a process called Fc fusion, Alprolix is the first haemophilia therapy to show prolonged circulation in the body, which has been shown in adults and adolescents with haemophilia to extend the time between prophylactic infusions.
Biogen Idec chief executive officer George Scangos said FDA approval of Alprolix is a significant milestone for the hemophilia B community.
"Alprolix offers people with hemophilia B the ability to prevent or reduce bleeding episodes with prophylactic infusions starting at least a week apart," Scangos said.
"We believe this new therapy will help more people with hemophilia and their caregivers realise the benefits of this treatment approach."
The company said that the National Hemophilia Foundation (NHF) recommends routine prophylaxis as optimal for the treatment of people with severe haemophilia.
The clinical studies have showed that people with severe haemophilia who follow this type of regimen experience fewer bleeding episodes and their related risks.
Hospital of the University of Pennsylvania assistant professor of medicine Patrick Fogarty said: "Alprolix addresses a critical need by allowing people with hemophilia B to maintain factor levels with prophylactic infusions once weekly or once every ten days."
The FDA approval is based on results from the global, Phase III B-LONG trial, as well as interim pharmacokinetic and safety data from the Phase III Kids B-LONG trial.
Results from the B-LONG trial showed that adults and adolescents with severe hemophilia B achieved prevention or reduction of bleeding episodes with prophylactic infusions at least a week apart.
The trial included two prophylaxis regimens; the weekly prophylaxis arm and the individualised-interval prophylaxis arm, when the dosing interval started at once every ten days.
Health Canada recently approved Alprolix for treatment of haemophilia B, while the drug is currently under review by regulatory authorities in several other countries, including Australia and Japan.
Common adverse reactions of Alprolix observed in the B-LONG trial were headaches and oral paresthesia.
Alprolix is expected to be commercially available to people with haemophilia B in the US in early May 2014.