FDA accepts for review CSL Behring BLA for CSL 627 (rVIIISingleChain) to treat haemophilia A

CSL Limited announced that the FDA has accepted for review CSL Behring’s Biologics License Application (BLA) for its novel investigational recombinant factor VIII single-chain, CSL 627 (rVIIISingleChain) for the treatment of haemophilia A.

In the pivotal clinical trial, rVIIISingleChain met all primary endpoints. Haemophilia A is a congenital bleeding disorder characterized 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 condition affects approximately 1 in 6,000 male births. The BLA submission is based on the AFFINITY clinical development programme, which includes a phase I/III open-label, multi-center trial examining safety and efficacy. The pharmacokinetics of rVIIISingleChain compared with recombinant human antihaemophilic factor VIII (octocog alfa, Advate) was also studied.

Comment: rVIIISingleChain is the only recombinant singlechain factor VIII product in late-stage development for the management of haemophilia A..