CSL Behring Announces First Two Patients Treated with HEMGENIX® (etranacogene dezaparvovec) Gene Therapy for Hemophilia B in Europe
2024年7月4日 - 4:00PM
CSL Behring Announces First Two Patients Treated with HEMGENIX®
(etranacogene dezaparvovec) Gene Therapy for Hemophilia B in
Europe
Global biotechnology leader CSL Behring (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 France2, 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 17th 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 CSLCSL (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. For more information about CSL,
visit CSL.com.
Media ContactsStephanie FuchsMobile: +49 151
584 388 60Email: Stephanie.Fuchs@cslbehring.com
References
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.