CSL's HEMGENIX® is a one-time,
single dose treatment for adults with hemophilia B who require
routine prophylaxis
OTTAWA,
ON, Oct. 26, 2023 /CNW/ -- Global
biotechnology leader CSL (ASX: CSL) today announced that
Health Canada has authorized HEMGENIX® (etranacogene
dezaparvovec), the first and only gene therapy for the treatment of
hemophilia B. HEMGENIX is indicated for the treatment of adults
with hemophilia B who require routine prophylaxis to prevent or
reduce the frequency of bleeding episodes. There is no clinical
experience of HEMGENIX use in patients with mild or moderate
hemophilia B (FIX activity >2%).
The approval is based on results from the ongoing Phase III,
open label, single-dose, single-arm, multi-center HOPE-B trial of
54 participants, the largest gene therapy trial in hemophilia B to
date. Data from the pivotal trial show that people with hemophilia
B treated with a single infusion of HEMGENIX demonstrated
significant increases in mean FIX activity levels (as measured by
one-stage assay) of 36.9% at 18-months, that were sustained at
36.7% at 24-months post-treatment, compared to the six-month lead
in period. Seven to 18 months post-infusion, the mean ABR was
reduced by 58% compared to the six-month lead-in period (4.13 to
1.73). Following infusion of HEMGENIX, 96% of patients (52 out
of 54) discontinued use of prophylaxis and remained free of
previous continuous routine prophylaxis therapy. Of the adverse
events reported based on phase 2b and
phase 3 trial (Hope-B), most frequently reported adverse drug
reactions were ALT elevations, headache, influenza-like illness and
AST elevations.
"This approval continues to demonstrate CSL's promise to
pursue, develop and deliver new innovative treatment options that
meet the needs of the rare disease community," said Dr.
Bill Mezzanotte, Head of Research
and Development, CSL. "We believe HEMGENIX has the potential
to change the treatment paradigm for people living with hemophilia
B and the healthcare professionals who treat them, as it addresses
the cause of the condition – faulty factor IX gene expression."
Hemophilia B is a rare, lifelong bleeding disorder caused by a
single gene defect, resulting in insufficient production of factor
IX, a protein primarily produced by the liver that helps the blood
to properly clot. Treatments for hemophilia B include prophylactic
infusions of factor IX replacement therapy 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. Those with hemophilia B may
also still experience spontaneous bleeding episodes as well as
limited mobility, joint damage, or severe pain as a result of the
disease. HEMGENIX allows people living with hemophilia B to
produce their own factor IX, which can lower the risk of
bleeding.
"The approval of HEMGENIX in Canada marks an important milestone and we
look forward to collaborating with the hemophilia B community to
provide access to this innovative treatment option," said
Philippe Hebert, General Manager
Canada, CSL Behring. "We are proud
to add this treatment to our portfolio of coagulation therapies and
look forward to patients benefiting from this therapy."
HEMGENIX® was also approved by the U.S. Food and
Drug Administration, and was granted conditional marketing
authorization by the European Commission (EC) for the European
Union and European Economic Area, and the United Kingdom's Medicines and Healthcare
products Regulatory Agency (MHRA). The multi-year clinical
development of HEMGENIX® was led by uniQure (Nasdaq:
QURE) and sponsorship of the clinical trials transitioned to CSL
after it licensed global rights to commercialize the treatment.
About Hemophilia B
Hemophilia B is a life-threatening rare disease caused by a
mutation on the F9 gene, resulting in low levels of functional
clotting factor IX. 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 53 patients
completing at least 18 months 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 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 tumor
characterization and vector integration analysis. No inhibitors to
factor IX were reported.
Long-term 24-month data presented at the 54th
American Society of Hematology (ASH) 2022 Annual Meeting and
Exposition and at The European Association for Haemophilia and
Allied Disorders (EAHAD) 2023 Annual Meeting 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.
Important Information for Canada
For more information and a
complete risk/benefit profile, please refer to the Product
Monograph available here.
About CSL
CSL (ASX:CSL; USOTC:CSLLY) is a leading
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, dialysis 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 30,000 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
CSLBehring.com/Vita and follow us on Twitter.com/CSL.
For more information about CSL, visit www.CSL.com.
Media Contacts
Maria
Tortoreto
Mobile: +1 201-248-5208
Email: maria.tortoreto@cslbehring.com
Etanjalie Ayala
Mobile: +1 610 297 1069
Email: etanjalie.ayala@cslbehring.com
In Australia
Kim O'Donohue
Mobile: +61 449 884 603
Email: kim.odonohue@csl.com.au
Jimmy Baker
Mobile: +61 450 909 211
Email: Jimmy.Baker@csl.com.au
SOURCE CSL