CSL (ASX:CSL) announced today that the US FDA has accepted for review the company’s Biologics License Application (BLA) for its novel investigational recombinant factor VIII single-chain (rVIIISingleChain) for the treatment of haemophilia A.
According to the company, in a pivotal clinical trial, rVIIISingleChain met all primary endpoints.
Haemophilia A is a congenital bleeding disorder characterised by deficient or defective factor VIII.
People with haemophilia A may experience prolonged or spontaneous bleeding, especially into the muscles, joints, or internal organs, the company said.
The condition affects approximately 1 in 6,000 male births.
“CSL Behring has one of the industry’s largest portfolios of biotherapies that improve the care and well-being of patients with a bleeding disorder,” said Dr Andrew Cuthbertson Chief Scientific Officer and Director of R&D, CSL. “Our scientific expertise and relationship with the bleeding disorders community led us to seek further advancements in the care and treatment of haemophilia. Today, we have the only recombinant single chain factor VIII product in late-stage development for the management of haemophilia A and we are excited to be one step closer to providing this innovative treatment to patients in the US.”
According to the company, the BLA submission is based on the AFFINITY clinical development program, which includes a phase I/III open-label, multi-centre trial examining safety and efficacy.