- HEMGENIX®, the first and only FDA approved gene
therapy for hemophilia B, has been proven to elevate and sustain
factor IX levels for years, significantly reduce the rate of annual
bleeds versus standard of care, and reduce or eliminate the need
for prophylactic treatment in 94% of patients (51 out of 54) after
a one-time infusion
KING OF
PRUSSIA, Pa., June 20,
2023 /PRNewswire/ -- Global biotechnology
leader CSL Behring today announced that the first patient has
received U.S. Food and Drug Administration (FDA) approved
HEMGENIX® (etranacogene dezaparvovec-drlb) for
hemophilia B in the United States.
HEMGENIX is the first and only gene therapy for the treatment of
adults with hemophilia B who currently use factor IX prophylaxis
therapy, or have current or historical life-threatening bleeding,
or have repeated, serious spontaneous bleeding episodes.
"As part of our longtime promise to patients, CSL Behring
is thrilled to mark this latest milestone of the first commercial
patient having received HEMGENIX and look forward to
continuing to deliver this paradigm-shifting treatment to the
hemophilia B community," said Bob
Lojewski, Senior Vice President and General Manager,
North America, CSL Behring. "To
the advocacy organizations, medical experts, healthcare
professionals, payers, channel partners, and the entire hemophilia
community who have collaborated with us to establish access for
this innovative therapy, we thank you and look forward to many more
patients benefiting from this therapy."
While prophylactic hemophilia B treatments have significantly
advanced over the past several decades and are effective,
treatments are typically administered on a strict infusion schedule
and breakthrough bleeds into joints are still
possible. HEMGENIX has the potential to fundamentally
transform the treatment paradigm through a single, one-time
infusion in people living with hemophilia B by addressing the
genetic cause of the condition.
In clinical trials, HEMGENIX demonstrated the ability to
elevate and sustain factor IX levels for years, significantly
reduce the rate of annual bleeds versus standard of care, and
reduce or eliminate the need for prophylactic treatment in 94% of
people who received the therapy. In addition to the potential
long-term health benefits from greater bleed protection and
liberation from infusion schedules, HEMGENIX could also generate
significant cost savings for the healthcare system at-large.
Healthcare costs can be 25 times higher for a person living with
hemophilia B compared to individuals who do not have a bleeding
disorder—amounting to a total adult lifetime cost of more than
$20 million per person.
"Discussions between CSL Behring and the payer community on
the value proposition offered for HEMGENIX have been positive,
which is reflected in the policies that have been written," added
Lojewski. "As of May 2023, payers
covering roughly 60 percent of the U.S. population have established
clear medical policies covering HEMGENIX. For those who have not
yet established policies, we anticipate that coverage decisions for
HEMGENIX will be reviewed on a case-by-case basis."
"HEMGENIX is an important addition to the current treatment
paradigm and a long-awaited advancement for the hemophilia B
community," said Matthew Ryan, MD,
Adult Hematologist and Medical Director at Hemophilia Outreach
Center, Green Bay, WI. "This
exciting treatment offers patients a chance to live a life without
frequent bleeds or the burden of regular infusions. The
availability of this new one-time treatment also sparks additional
conversations in the community when it comes to treatment options
and goals, which is extremely important when managing a life-long
condition, as an individual's needs can change for a variety of
reasons."
People living with hemophilia B who have decided with their
doctor to move forward with treatment with HEMGENIX, can enroll in
the HEMGENIX ConnectSM program where they will be
assigned a dedicated support team, including a Patient Resource
Navigator and CSL Case Manager, who can assist with questions about
gene therapy, the treatment journey and insurance coverage.
In further demonstration of the company's commitment to the
hemophilia B community, CSL Behring is providing ongoing
training to centers who may administer HEMGENIX and encouraging
long term data collection through the ATHN registry. Registry data
will be informative to all stakeholders and will generate
additional evidence on the long-term safety, efficacy, and
durability of gene therapy.
HEMGENIX was also approved by the European Commission (EC)
for the European Union and European Economic Area and was granted
conditional marketing authorization by the United Kingdom's Medicines and Healthcare
product Regulatory Agency. 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 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 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 Safety Information (ISI)
What is HEMGENIX®?
HEMGENIX®, etranacogene
dezaparvovec-drlb, is a one-time gene therapy for the treatment of
adults with hemophilia B who:
- Currently use Factor IX prophylaxis therapy, or
- Have current or historical life-threatening bleeding, or
- Have repeated, serious spontaneous bleeding episodes.
HEMGENIX® is administered as a single
intravenous infusion and can be administered only once.
What medical testing can I expect to be given before and
after administration
of HEMGENIX®?
To determine
your eligibility to receive HEMGENIX®, you will be
tested for Factor IX inhibitors. If this test result is positive, a
retest will be performed 2 weeks later. If both tests are positive
for Factor IX inhibitors, your doctor will not administer
HEMGENIX® to you. If, after administration of
HEMGENIX®, increased Factor IX activity is not achieved,
or bleeding is not controlled, a post-dose test for Factor IX
inhibitors will be performed.
HEMGENIX® may lead to elevations of liver
enzymes in the blood; therefore, ultrasound and other testing will
be performed to check on liver health before
HEMGENIX® can be administered. Following
administration of HEMGENIX®, your doctor will monitor
your liver enzyme levels weekly for at least 3 months. If you have
preexisting risk factors for liver cancer, regular liver health
testing will continue for 5 years post-administration. Treatment
for elevated liver enzymes could include corticosteroids.
What were the most common side effects
of HEMGENIX® in clinical
trials?
In clinical trials for HEMGENIX®, the
most common side effects reported in more than 5% of patients were
liver enzyme elevations, headache, elevated levels of a certain
blood enzyme, flu-like symptoms, infusion-related reactions,
fatigue, nausea, and feeling unwell. These are not the only side
effects possible. Tell your healthcare provider about any side
effect you may experience.
What should I watch for during infusion
with HEMGENIX®?
Your doctor
will monitor you for infusion-related reactions during
administration of HEMGENIX®, as well as for at least 3
hours after the infusion is complete. Symptoms may include chest
tightness, headaches, abdominal pain, lightheadedness, flu-like
symptoms, shivering, flushing, rash, and elevated blood pressure.
If an infusion-related reaction occurs, the doctor may slow or stop
the HEMGENIX® infusion, resuming at a lower
infusion rate once symptoms resolve.
What should I avoid after
receiving HEMGENIX®?
Small
amounts of HEMGENIX® may be present in your blood,
semen, and other excreted/secreted materials, and it is not known
how long this continues. You should not donate blood, organs,
tissues, or cells for transplantation after receiving
HEMGENIX®.
Please see full prescribing
information for
HEMGENIX®.
You are encouraged to report negative side effects of
prescription drugs to the FDA. Visit www.fda.gov/medwatch, or
call 1-800-FDA-1088.
You can also report side effects to CSL Behring's
Pharmacovigilance Department at 1-866-915-6958.
About CSL Behring
CSL Behring is a global
biotherapeutics leader driven by our promise to save lives. Focused
on serving patients' needs by using the latest technologies, we
discover, develop and deliver innovative therapies for people
living with conditions in the immunology, hematology,
cardiovascular and metabolic, respiratory, and transplant
therapeutic areas. We use three strategic scientific platforms of
plasma fractionation, recombinant protein technology, and cell and
gene therapy to support continued innovation and continually refine
ways in which products can address unmet medical needs and help
patients lead full lives.
CSL Behring operates one of the world's largest plasma
collection networks, CSL Plasma. Our parent
company, CSL (ASX:CSL; USOTC:CSLLY), headquartered in
Melbourne, Australia, employs
32,000 people, and delivers its lifesaving therapies to people in
more than 100 countries. For inspiring stories about the promise of
biotechnology, visit CSLBehring.com/Vita and follow us
on Twitter.com/CSLBehring.
Media Contact
Etanjalie Ayala
Mobile: +1 610 297 1069
Email: etanjalie.ayala@cslbehring.com
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