MARBURG, Germany, July 04, 2024 (GLOBE NEWSWIRE) -- Global biotechnology leader
CSL Behring (https://www.csl.com/) (ASX: CSL) today announced that two
hemophilia B patients were treated with the gene therapy HEMGENIX(®)
(etranacogene dezaparvovec) at Hemophilia Treatment Centers in France. This
milestone achievement makes HEMGENIX(®) the first gene therapy administered as a
treatment in a real-world setting for hemophilia B in Europe.
HEMGENIX(®) is the first one-time gene therapy approved in Europe for the
treatment of adults with severe and moderately severe hemophilia B, an inherited
bleeding disorder caused by the lack of Factor IX (a protein needed to produce
blood clots to stop bleeding). It is used in adults without a history of Factor
IX inhibitors.(1)
Following European Commission approval, HEMGENIX(®) was the first ever therapy
to be granted Direct Access in France(2), thus enabling the first patients to be
treated in Europe outside of the clinical program.
Though effective, current therapies can be time intensive and require regular
treatment that can have a substantial impact on a patient's daily life.(3)
HEMGENIX(®) offers a one-time treatment, allowing people living with hemophilia
B to produce their own Factor IX, which can lower the risk of bleeding.(4)
"Only a few decades ago, gene therapy for hemophilia was a distant concept,
which has now become reality. Accordingly, the first two patients treated with
HEMGENIX(®) since receiving European approval is a major accomplishment and a
testament to the joint commitment of the hemophilia B community, as well as the
access and reimbursement authorities, in bringing innovative therapies to
patients," said Dr Lutz Bonacker SVP and General Manager, CSL Behring Commercial
Operations Europe. "This milestone has been made possible by the innovative
Direct Access scheme adopted in France, allowing patients to benefit from early
access to pioneering treatments. We are encouraged to see increasing access to
gene therapies in European countries and are fully committed to ensuring that
access to potentially life-changing treatment continues."
HEMGENIX(®) was granted conditional marketing authorisation by the European
Commission (EC) for the European Union and European Economic Area in February
2023, following approval from the U.S. Food and Drug Administration (FDA) in
November 2022. It has also been approved by Health Canada, the United Kingdom's
Medicines and Healthcare products Regulatory Agency (MHRA), Switzerland's
Swissmedic and Australia's Therapeutic Goods Administration (TGA).
The multi-year clinical development of HEMGENIX(®) was led by uniQure and
sponsorship of the clinical trials transitioned to CSL after it licensed global
rights to commercialise the treatment.
About Hemophilia B
Hemophilia B is a life-threatening rare disease. People with the condition are
particularly vulnerable to bleeds in their joints, muscles, and internal organs,
leading to pain, swelling, and joint damage. Current treatments for moderate to
severe hemophilia B include life-long prophylactic infusions of factor IX to
temporarily replace or supplement low levels of the blood-clotting factor.
About HEMGENIX(®)
HEMGENIX(®) is a gene therapy that reduces the rate of abnormal bleeding in
eligible people with hemophilia B by enabling the body to continuously produce
factor IX, the deficient protein in hemophilia B. It uses AAV5, a non-infectious
viral vector, called an adeno-associated virus (AAV). The AAV5 vector carries
the Padua gene variant of Factor IX (FIX-Padua) to the target cells in the
liver, generating factor IX proteins that are 5x-8x more active than normal.
These genetic instructions remain in the target cells, but generally do not
become a part of a person's own DNA. Once delivered, the new genetic
instructions allow the cellular machinery to produce stable levels of factor IX.
About the Pivotal HOPE-B Trial
The pivotal Phase III HOPE-B trial is an ongoing, multinational, open-label,
single-arm study to evaluate the safety and efficacy of HEMGENIX(®). Fifty-four
adult hemophilia B patients classified as having moderately severe to severe
hemophilia B and requiring prophylactic factor IX replacement therapy were
enrolled in a prospective, six-month or longer observational period during which
time they continued to use their current standard of care therapy to establish a
baseline Annual Bleeding Rate (ABR). After the six-month lead-in period,
patients received a single intravenous administration of HEMGENIX(®) at the
2x10^13 gc/kg dose. Patients were not excluded from the trial based on pre-
existing neutralizing antibodies (NAbs) to AAV5.
A total of 54 patients received a single dose of HEMGENIX(®) in the pivotal
trial, with 52 patients completing at least three years of follow-up. The
primary endpoint in the pivotal HOPE-B study was ABR 52 weeks after achievement
of stable factor IX expression (months 7 to 18) compared with the six-month
lead-in period. For this endpoint, ABR was measured from month seven to month
18 after infusion, ensuring the observation period represented a steady-state
factor IX transgene expression. Secondary endpoints included assessment of
factor IX activity.
No serious treatment-related adverse reactions were reported. One death
resulting from urosepsis and cardiogenic shock in a 77-year-old patient at 65
weeks following dosing was considered unrelated to treatment by investigators
and the company sponsor. A serious adverse event of hepatocellular carcinoma was
determined to be unrelated to treatment with HEMGENIX(®) by independent
molecular tumour characterization and vector integration analysis. No inhibitors
to factor IX were reported.
Long-term three-year data presented at the 17(th) Annual Congress of the
European Association for Haemophilia and Allied Disorders (EAHAD) 2024 continue
to reinforce the potential long-lasting efficacy and safety of HEMGENIX(®) and
the ongoing benefit of this treatment for people living with hemophilia B.
About CSL
CSL (https://www.csl.com/) (ASX:CSL; USOTC:CSLLY) is a global biotechnology
company with a dynamic portfolio of lifesaving medicines, including those that
treat hemophilia and immune deficiencies, vaccines to prevent influenza, and
therapies in iron deficiency and nephrology. Since our start in 1916, we have
been driven by our promise to save lives using the latest technologies. Today,
CSL - including our three businesses: CSL Behring, CSL Seqirus and CSL Vifor -
provides lifesaving products to patients in more than 100 countries and employs
32,000 people. Our unique combination of commercial strength, R&D focus and
operational excellence enables us to identify, develop and deliver innovations
so our patients can live life to the fullest. For inspiring stories about the
promise of biotechnology, visit CSL.com/Vita (https://www.csl.com/we-are-
csl/vita-original-stories). For more information about CSL, visit CSL.com
(https://www.csl.com/).
Media Contacts
Stephanie Fuchs
Mobile: +49 151 584 388 60
Email: Stephanie.Fuchs@cslbehring.com (mailto:Stephanie.Fuchs@cslbehring.com)
References
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(1) European Medicines Agency. First Gene therapy to treat haemophilia B.
Available at: https://www.ema.europa.eu/en/news/first-gene-therapy-treat-
haemophilia-b. (Accessed May 2024).
(2) Republique Française. Légifrance: Article 62 of Law No. 2021-1754. Available
at: https://www.legifrance.gouv.fr/jorf/id/JORFTEXT000048551003 (Accessed May
2024).
(3) Leebeek, F & Miesbach, W. (2021) Gene therapy for haemophilia: a review on
clinical benefit, limitations, and remaining issues. Blood. Vol 138, Issue 11.
pp923-931.
(4) Coppens M et al. Etranacogene dezaparvovec gene therapy for haemophilia B
(HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm,
multicentre, phase 3 trial. The Lancet Haematology 2024; 11(4):E265-E275.
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