FDA approves first long-acting recombinant coagulation Factor IX concentrate for patients with Hemophilia B

FDA has approved Coagulation Factor IX (Recombinant), Fc Fusion Protein (Alprolix, Biogen Idec), for use in adults and children who have hemophilia B; it also granted the product orphan-drug designation. Alprolix is the first hemophilia B treatment designed for less frequent injections when used to prevent or reduce bleeding frequency.

Alprolix is approved to help control and prevent bleeding episodes, manage bleeding during surgical procedures, and prevent or reduce the frequency of bleeding episodes (prophylaxis). Alprolix consists of the Factor IX molecule linked to Fc, a protein fragment that is found in antibodies. This makes the product last longer in circulation.

The newly approved product is the first significant advance in hemophilia B treatment in more than 17 years.

A multicenter clinical trial evaluated the safety and efficacy of Alprolix, comparing each of 2 prophylactic treatment regimens to on-demand treatment. A total of 123 individuals with severe hemophilia B, ages 12-71, were followed for up to a year and a half. The studies demonstrated the effectiveness of Alprolix in the prevention and treatment of bleeding episodes and during perioperative management of patients undergoing a surgical procedure. No safety concerns were identified in this trial.

Across the routine prophylaxis and on-demand therapy arms, 8.4% of participants reported adverse reactions that included headache, oral paresthesia (abnormal sensation in the mouth), dizziness, dysgeusia (taste alteration), breath odor, fatigue, infusion-site pain, palpitations, obstructive uropathy (an obstruction caused by a clot in the urinary collecting system) and hypotension (low blood pressure). Each event occurred in 2 or fewer study participants.

“We believe the clinical value of Alprolix includes its ability to reduce bleeding episodes with a favorable safety and tolerability profile,” said Andrew Law, Biogen Idec spokesperson. “These data and the ability to enable prophylactic infusions starting at least a week apart represent an important new option.

The National Hemophilia Foundation recommends a prophylactic regimen as optimal therapy for people with severe hemophilia. Studies show that patients with severe hemophilia who follow a prophylactic infusion schedule experience fewer bleeding episodes and associated risks.

Hemophilia B is a rare, chronic, inherited disorder in which the clotting function of the blood is impaired, which can lead to recurrent and extended bleeding episodes.

Hemophilia B is a rare, chronic, inherited disorder in which the clotting function of the blood is impaired, which can lead to recurrent and extended bleeding episodes.

 Hemophilia B is a rare, chronic, inherited disorder in which the clotting function of the blood is impaired, which can lead to recurrent and extended bleeding episodes.