The US Food and Drug Administration has approved CSL Behring’s Idelvion, paving the way for patients with Haemophilia B to access treatment with the first and only factor IX therapy that offers up to 14-day dosing to protect against or reduce bleeds.
Haemophilia B is a rare inherited disorder that prevents blood from clotting normally, leaving patients at risk of potentially serious bleeding. Idelvion is used to replace Factor IX, a naturally occurring clotting factor that is missing or defective in people with the condition.
The drug has been cleared for use in both children and adults with haemophilia B for routine prophylaxis to prevent or reduce the frequency of bleeding episodes; on-demand control and prevention of bleeding episodes; and the perioperative management of bleeding.
“The approval of this long-acting recombinant factor IX therapy for haemophilia B is vital, as physicians need more options to help their patients effectively and safely manage their bleeding disorder,” said Elena Santagostino, Professor in the Medical School of Clinical and Experimental Haematology at the University of Milan/IRCCS Maggiore Hospital, and lead investigator of the PROLONG-9FP clinical development programme.
Idelvion provides patients “with greater freedom from frequent infusions which is an important attribute for patients who require a prophylactic regimen but don’t want treatment to disrupt their active lives,” she noted.
The company said it is expecting to launch the drug in the US later this month (March).
Idelvion is currently approved in Canada. The European Medicines Agency’s Committee for Medicinal Products for Human Use recently endorsed its marketing authorisation in the European Union, while the drug is also under review in Australia, Switzerland and Japan.