The European Commission has granted conditional marketing authorisation for CSL's HEMGENIX (etranacogene dezaparvovec) as the first and only one-time gene therapy for treating severe and moderately severe haemophilia B (congenital Factor IX deficiency) in adults without a history of Factor IX inhibitors.
The company said that in an ongoing clinical trial, HEMGENIX reduced the rate of annual bleeds with a single infusion by delivering a functional gene that acts as a blueprint for coagulation Factor IX.
The conditional marketing authorisation of HEMGENIX in Europe follows its approval in the US late last year. It is currently under review by the UK's Medicines and Healthcare products Regulatory Agency.
CSL said it is preparing a dossier for submission to Australia's TGA.
“The approval of HEMGENIX in Europe is the essence of great science delivering a medicine that we believe can transform the treatment paradigm for both people living with hemophilia B and the healthcare professionals who treat them,” said Dr Bill Mezzanotte, CSL's head of research and development and chief medical officer.
“HEMGENIX, and our partnership with uniQure, underscore CSL’s promise to pursue, develop and deliver disruptive innovations when patients can benefit, particularly in disease states we know well like hemophilia B.”
People living with hemophilia B currently require lifelong treatment of intravenous infusions of Factor IX to maintain sufficient levels.
In a clinical trial, patients treated with HEMGENIX demonstrated stable and durable increases in mean Factor IX activity levels, which led to an adjusted annualized bleed rate (ABR) reduction of 64 per cent.
Following infusion of HEMGENIX, 96 per cent of patients discontinued routine Factor IX prophylaxis, and mean Factor IX consumption was reduced by 97 per cent at 18 months post-treatment, compared to the lead-in period.
“At CSL Behring, our promise is simple - to save and improve lives - and we achieve this by living our core values of patient focus, innovation, integrity, collaboration, and superior performance,” said Lutz Bonacker, the senior vice president and general manager of Commercial Operations for CSL Behring in Europe.
“The approval of HEMGENIX in Europe is a result of that focus and a milestone for the hemophilia B community, and we now need to work to ensure that as many eligible patients across Europe can access this innovative treatment as possible. We are fully committed to working together with payers and other stakeholders to achieve this.”