Pharma Focus Asia

European Commission Approved CSL's HEMGENIX®, the First Gene Therapy for Hemophilia B

Monday, February 20, 2023

Global biotechnology leader CSL announced that the European Commission has granted conditional marketing authorisation (CMA) for HEMGENIX® (etranacogene dezaparvovec), the first and only one-time gene therapy for the treatment of severe and moderately severe hemophilia B (congenital Factor IX deficiency) in adults without a history of Factor IX inhibitors. In the 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, a protein important for blood clotting. It is the first approved gene therapy for hemophilia B in the European Union (EU) and European Economic Area (EEA).

HEMGENIX® is an adeno-associated virus five (AAV5)-based gene therapy given as a one-time treatment for moderately severe to severe hemophilia B patients. HEMGENIX® (also known as CSL222, previously known as AMT-061) uses a specific type of AAV, called AAV5, as its vector. The AAV5 vector carries the naturally occurring Padua gene variant of Factor IX (Factor IX-Padua), which generates Factor IX proteins that are 5x-8x more active than normal.

The approval of HEMGENIX® in Europe is the essence of great science delivering a medicine which can transform the treatment paradigm for both people living with hemophilia B and the healthcare professionals who treat them. HEMGENIX® underscores CSL's promise to deliver life-changing innovations having the potential to help patients lead full lives.

This approval marks an important step forward in the treatment of hemophilia B, which changes the lives of people weakened by bleeding in the muscles, joints and internal organs, alleviating the burden of lifelong intravenous infusions of Factor IX products. Data from the HOPE-B study demonstrated the potential of HEMGENIX® to remove the need for routine prophylaxis, by providing durable Factor IX activity, as well as improved bleeding outcomes and quality of life for people with hemophilia B.

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