Etranacogene Dezaparvovec, Marketed as Hemgenix, Approved in EU for Hemophilia B


The gene therapy was approved in the US in the same indication in November 2022.

Etranacogene dezaparvovec (Hemgenix; CSL Behring) has been approved by the European Commission (EC) for the treatment of severe and moderately severe hemophilia B in adults without a history of Factor IX inhibitors.1

"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," Bill Mezzanotte, MD, head, research & development, and chief medical officer, CSL, said in a statement.1 "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.

The EC’s decision follows the Committee for Medicinal Products for Human Use’s positive opinion in December 2022 and is based on continued positive data generated by the phase 3 HOPE-B trial (NCT03569891). The trial had previously met its primary end point of reduction in annualized bleeding rate (ABR) post-treatment compared with baseline factor IX (FIX) prophylactic therapy.

READ MORE: Hemgenix Approval May Pave Way for Val-Rox in Hemophilia A

“From month 18 to month 24 of follow-up, we're seeing stable FIX expression and no new adverse events,” investigator Steven Pipe, MD, professor, pediatric hematology/oncology at CS Mott Children’s Hospital, previously told CGTLive at the 64th American Society of Hematology (ASH) Annual Meeting, held December 10-12, 2022, where new follow-up data were presented that continue to support the therapy’s durability.

Hemgenix was approved in the US under the same indication in November 2022. Data from the HOPE-B trial demonstrated that participants had a mean FIX activity of 39.0 IU/dL (standard deviation [SD], 18.7; range, 8.2-97.1) at 6 months post-treatment and 36.9 IU/dL (SD, 21.4; range, 4.5-122.9) at 18 months.2 The 52-week adjusted ABR for all bleeds was reduced by 64% (= .0002) from 4.19 during the ≥ 6-month lead-in period to 1.51 during months 7-18 and there was an overall 97% reduction in mean unadjusted annualized FIX consumption.

"This approval marks an important step forward in the treatment of hemophilia B, which could be transformative for people who are debilitated by bleeds into their muscles, joints and internal organs, alleviating the burden of lifelong intravenous infusions of Factor IX products," Wolfgang Miesbach, MD, PhD, head, Coagulation Disorders, Comprehensive Care Center, University Hospital of Frankfurt, added to the statement.1 "Data from the HOPE-B study demonstrate 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."

1. First Gene Therapy for Hemophilia B, CSL's HEMGENIX®, Approved by the European Commission. News release. CSL Behring. February 20, 2023.
2. uniQure and CSL Behring announce primary endpoint achieved in HOPE-B pivotal trial of Etranacogene Dezaparvovec gene therapy in patients with Hemophilia B. News release. UniQure. December 9, 2021.
3. FDA approves first gene therapy to treat adults with hemophilia B. News release. FDA. November 22, 2022.
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