Hemophilia B

Hero hemophilia b

The world’s first gene therapy for hemophilia B 

In November 2022, the U.S. Food and Drug Administration (FDA) approved HEMGENIX@ (etranacogene dezaparvovec), the world’s gene therapy for hemophilia B. This is uniQure’s second internally-developed gene therapy to achieve approval and the world’s first gene therapy for hemophilia B, an historic achievement based on more than a decade of research and clinical development. 

In February 2023, HEMGENIX received conditional marketing authorization (CMA) from the European Commission, making it the first and only one-time gene therapy for hemophilia in the EU.

Hemophilia B is a rare, lifelong bleeding disorder caused by a single gene mutation, resulting in the absence or deficiency in the production of factor IX, a protein primarily produced by the liver that helps blood clots form during the healing process. Treatments for hemophilia B include prophylactic infusions of factor IX to temporarily replace or supplement low levels of blood-clotting factor and, while these therapies are effective, those with hemophilia B must adhere to strict, lifelong infusion schedules. They may also still experience spontaneous bleeding episodes as well as limited mobility, joint damage or severe pain as a result of the disease.

For appropriate patients, etranacogene dezaparvovec allows people living with hemophilia B to produce their own factor IX, which can lower the risk of bleeding. In clinical trials, etranacogene dezaparvovec reduced the rate of annual bleeds and the majority of patients treated discounted use of prophylactic treatment after a one-time infusion.

uniQure led the research and clinical development of etranacogene dezaparvovec, which included three clinical trials across 34 global sites and involving 67 adults with hemophilia B.

The Advantages of AAV5 in Gene Therapy

AAV5-based gene therapies have been demonstrated to be safe and well-tolerated in a multitude of clinical trials, including five uniQure trials conducted in nearly 80 patients in hemophilia B and other indications. No patient treated in clinical trials with uniQure’s AAV5 gene therapies has experienced any confirmed cytotoxic T‑cell-mediated immune response to the capsid. Additionally, pre-clinical and clinical data show that AAV5-based gene therapies may be viable treatments in patients with pre-existing antibodies to AAV5, thereby potentially increasing patient eligibility for treatment compared to other gene therapy product candidates.

Licensing Agreement with CSL Behring

In May 2021, uniQure and CSL completed a licensing transaction providing CSL Behring with exclusive rights to commercialize HEMGENIX globally. uniQure is responsible for the global manufacturing of HEMGENIX at its licensed Lexington, MA facility. Under the terms of the agreement, uniQure has received payments from CSL totaling $500 million, and is eligible to receive up to an additional $1.6 billion in commercial milestone payments and tiered, double-digit royalties in a range up to a low-twenties percentage of net product sales arising from the collaboration.

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