First Gene Therapy for Hemophilia B Approved by the European Commission

This EC CMA received by HEMGENIX therapy for the treatment of adults with severe and moderately severe hemophilia B without a history of Factor IX inhibitors makes it the first ever one-time gene therapy approved for this disorder in the European Union (EU) and European Economic Area (EEA).

HEMGENIX’s approval follows results from the pivotal HOPE-B trail, the biggest hemophilia B trial to date, that led to the CHMP’s positive opinion in December 2022.

“This achievement is based on more than a decade of research and clinical development led by uniQure, and we are grateful for the tireless dedication of our employees, clinicians, patients and their families who made this possible,” said Matt Kapusta, chief executive officer of uniQure.

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The HOPE-B trial demonstrated that patients gained stable and durable increases in mean Factor IX activity levels and experienced a resultant reduction in adjusted annualized bleed rate (ABR) by 64% with HEMGENIX infusion.

Post-infusion 96% of patients in the trial discontinued Factor IX prophylaxis, with mean Factor IX consumption reduced by 97% after 18 months.

“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,” commented Professor Wolfgang Miesbach, Head of Coagulation Disorders at the Comprehensive Care Center, University Hospital of Frankfurt.

The 24-month HOPE-B analysis showed a sustained and durable response in patients, with no serious treatment-related adverse events reported.

Elsewhere in the world, HEMEGENIX was approved in the U.S by the FDA in November 2022, and it is currently still under review in the UK by The Medicines and Healthcare Products Regulatory Agency.

In total UniQure conducted three clinical trials of HEMGENIX, across 34 global sites involving 67 adults with hemophilia B.

The Company entered into a licensing agreement in May 2021 providing CSL with exclusive commercialization and clinical development rights.

UniQure maintains manufacturing responsibilities and receives commercial milestone payments, as well as royalties and a percentage of net sales.

 “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, Head of Research & Development and CMO, CSL.

Source: UniQure Press Release

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