The U.S. Food and Drugs Administration gave the green signal to Biogen's Alprolix, the first long-acting treatment for patients with Hemophilia B.
The drug can be used by both adults and children and is designed to reduce the need for injections during bleeding episodes, FDA revealed in a press release.
Alprolix is manufactured by Biogen and can be used to control and prevent bleeding episodes, manage bleeding during surgical procedures, and prevent or reduce the frequency of bleeding episodes. It also contains Factor IX molecules that are present in other antibiotics. The molecules make the drug last longer in circulation.
"The approval of this product provides another therapeutic option for the treatment and prevention of bleeding in patients with Hemophilia B," said Karen Midthun, M.D., director of the FDA's Center for Biologics Evaluation and Research, in a statement.
Hemophilia B is also known as the Christmas disease and is a hereditary bleeding disorder caused by a lack of blood clotting factor IX. Without enough factor IX, the blood cannot clot properly to control bleeding. It is caused by an inherited X-linked recessive trait, with the defective gene located on the X chromosome.
The severity of symptoms can vary, and the severe forms become apparent early on. Bleeding is the main symptom. Bleeding problems usually show up when the infant starts crawling and walking.
The safety and effectiveness of Alprolix were tested in a multi-center clinical trial. A total of 123 individuals with severe Hemophilia B, ages 12-71, were followed for up to a year and a half. The trial revealed that the drug was effective in preventing and treating bleeding episodes. It was also effective during perioperative management of patients undergoing a surgical procedure.
No safety concerns or side effects were identified in this trial.
