CSL Limited has wrapped up the acquisition of uniQure’s etranacogene dezaparvovec, a novel gene therapy for the treatment of haemophilia B.
Under the agreement, CSL will make an initial an upfront cash payment of US$450 million with further milestone payments and royalties to follow as the therapy is developed and commercialised.
Etranacogene dezaparvovec is currently in Phase 3 clinical trials and has been shown to result in near-normal levels of Factor IX (FIX) – the blood clotting protein lacking in people with haemophilia B.
uniQure will complete the Phase 3 trial and scale up manufacture for initial commercial supply while CSL Behring will be responsible for regulatory submissions and commercialisation.
CSL CEO and Managing Director Paul Perreault, said the acquisition complements both CSL Behring’s cell and gene therapy scientific platform and its haematology product portfolio, which include other treatments for haemophilia B as well as therapies for treating haemophilia A, von Willebrand disease, thrombosis, and other life-threatening conditions.
“We are continuing to build on our legacy of delivering lifesaving innovations in haematology with today’s news,” Mr Perreault said.
“This agreement enables us to take forward a gene therapy that, if approved, has the potential to transform the lives of haemophilia B patients.
“Etranacogene dezaparvovec has the potential to be the first-ever gene therapy approved for haemophilia B and help CSL Behring deliver on our ongoing commitment to improving the lives of those living with haemophilia B.”