- A one-time dose of BEQVEZ has reduced bleeds post-treatment
compared to standard of care with a median of zero bleeds (range 0
to 19) after up to three years of follow-up, providing sustained
bleed protection and potentially avoiding years of treatment burden
with prophylaxis for many patients
Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and
Drug Administration (FDA) has approved BEQVEZ™ (fidanacogene
elaparvovec-dzkt) for the treatment of adults with moderate to
severe hemophilia B who currently use factor IX (FIX) prophylaxis
therapy, or have current or historical life-threatening hemorrhage,
or have repeated, serious spontaneous bleeding episodes, and do not
have neutralizing antibodies to adeno-associated virus serotype
Rh74var (AAVRh74var) capsid as detected by an FDA-approved test.
BEQVEZ is a one-time treatment that is designed to enable people
living with hemophilia B to produce FIX themselves rather than the
current standard of care, which requires regular intravenous
infusions of FIX that are often administered multiple times a week
or multiple times a month.1,2
“Many people with hemophilia B struggle with the commitment and
lifestyle disruption of regular FIX infusions, as well as
spontaneous bleeding episodes, which can lead to painful joint
damage and mobility issues,” said Adam Cuker, M.D., M.S., Director,
Penn Comprehensive and Hemophilia Thrombosis Program. “A one-time
treatment with BEQVEZ has the potential to be transformative for
appropriate patients by reducing both the medical and treatment
burden over the long term.”
Hemophilia B is a rare genetic bleeding disorder that prevents
normal blood clotting because of a deficiency in FIX that causes
those with the disease to bleed more frequently and longer than
others.3,4 The standard of care for hemophilia B treatment is
prophylactic infusions of FIX replacement therapy that temporarily
replace or supplement low levels of blood-clotting factor.2,4
Despite prophylaxis and regular intravenous infusions, many people
living with moderate to severe hemophilia B are at risk of
spontaneous bleeding episodes.5,6,7 The current standard of care
also places strain on healthcare systems’ budgets and resource
utilization.6,8,9,10 According to the World Federation of
Hemophilia, more than 38,000 people worldwide are living with
hemophilia B.11
“This milestone is a testament to Pfizer’s continued effort to
advance the standard of care for people living with hemophilia,
with the delivery of a medicine that has the potential to offer
both long-term bleed protection and value to the healthcare system
because of its one-time administration,” said Aamir Malik, Chief
U.S. Commercial Officer and Executive Vice President, Pfizer. “We
are leveraging our expertise that comes with more than 40 years of
experience in the hemophilia space, and are proactively working
with treatment centers, payers, and the hemophilia community to
appropriately help ensure the healthcare system is prepared to
readily deliver BEQVEZ to the patients who can benefit from
it.”
With BEQVEZ now approved for use, Pfizer is launching an
innovative warranty program based on durability of patient response
to treatment. The goal of the warranty is to provide greater
certainty to payers, maximize access for eligible patients who
receive BEQVEZ, and offer financial protection by insuring against
the risk of efficacy failure.
“For people living with hemophilia, disease management can
interfere with many aspects of their lives. A one-time infusion of
BEQVEZ may allow eligible patients more time for the things they
love,” said Kim Phelan, Chief Operating Officer, The Coalition for
Hemophilia B. “We are excited to have BEQVEZ as a promising
treatment option for eligible people living with hemophilia B. We
look forward to learning more and celebrating with the community
and with Pfizer at our annual conference that is currently taking
place.”
BEQVEZ is currently under review with the European Medicines
Agency (EMA), and the treatment recently received regulatory
approval in Canada. In addition to BEQVEZ, Pfizer currently has two
other Phase 3 programs investigating gene therapy in populations
where there is a high unmet need: hemophilia A (giroctocogene
fitelparvovec) and Duchenne muscular dystrophy (fordadistrogene
movaparvovec). Additionally, a Phase 3 trial is investigating
marstacimab, a novel, investigational anti-tissue factor pathway
inhibitor for the treatment of people with hemophilia A and B with
and without inhibitors. A Biologics License Application and
European Marketing Authorization Application for marstacimab are
currently under review with the FDA and EMA, respectively.
About BEQVEZ (fidanacogene elaparvovec-dzkt) BEQVEZ is an
adeno-associated virus (AAV)-based gene therapy designed to
introduce in the transduced cells a functional copy of the FIX gene
encoding a high-activity FIX variant. For eligible patients living
with hemophilia B, the goal of this gene therapy is to enable them
to produce FIX themselves via this one-time treatment rather than
having to receive frequent infusions of FIX, as is the current
standard of care.1,2,4 It is currently approved in the U.S. for the
treatment of adults with moderate to severe hemophilia B
(congenital factor IX deficiency) who:
- Currently use factor IX prophylaxis therapy, or
- Have current or historical life-threatening hemorrhage, or
- Have repeated, serious spontaneous bleeding episodes, and,
- Do not have neutralizing antibodies to adeno-associated virus
serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved
test.
For eligible patients who are prescribed BEQVEZ, Pfizer offers
personalized patient support services including financial
assistance resources and logistical support through the Pfizer Gene
Together program. More information is available on
www.PfizerGeneTogether.com.
In December 2014, Pfizer licensed BEQVEZ from Spark
Therapeutics. Under the agreement, Pfizer assumed responsibility
for pivotal studies, any regulatory activities, and potential
global commercialization of this gene therapy.
About BENEGENE-2 The FDA approval is based on the results
from the pivotal BENEGENE-2 study, a Phase 3, open-label,
single-arm study to evaluate the efficacy and safety of BEQVEZ in
adult male participants (age 18–65) with moderately severe to
severe hemophilia B (defined as FIX circulating activity of 2% or
less). The main objective of the study is to evaluate the
annualized bleeding rate (ABR) for participants treated with gene
therapy versus FIX prophylaxis replacement regimen, administered as
part of usual care.
The study enrolled 45 participants. Eligible study participants
have completed a minimum of six months of routine FIX prophylaxis
therapy during the lead-in study (NCT03587116) and received a
single intravenous infusion of BEQVEZ at a dose of 5 x 1011 vg/kg
of body weight. Clinical trial participants will be followed for up
to a total of 15 years, including six years in the BENEGENE-2 study
and an additional nine years as part of a separate Phase 3 study
(NCT05568719) to learn about the long-term safety and efficacy of
BEQVEZ.
BENEGENE-2 met its primary endpoint of non-inferiority in the
ABR of total bleeds post-BEQVEZ infusion versus prophylaxis regimen
with FIX, administered as part of usual care. A mean ABR of 2.5 was
observed among patients who received BEQVEZ in the efficacy
evaluation period – defined as between week 12 and data cutoff
(median 1.8 years of follow-up) – after receiving the one-time dose
compared to a mean ABR of 4.5 during the lead-in pre-treatment
period of at least six months (median 1.2 years of follow-up).
Bleeds were eliminated in 60% of patients compared to 29% in the
prophylaxis arm. A median ABR of zero (range of 0 to 19) was
observed during the efficacy evaluation period compared to the
prophylaxis arm in which a median ABR of 1.3 (range of 0 to 53.9)
was observed.
BEQVEZ was generally well-tolerated in patients who received it.
The most common adverse reaction (incidence ≥5%) reported in Phase
3 and 1/2 clinical studies was an increase in transaminases. No
deaths, serious adverse events related to treatment or associated
with infusion reactions, thrombotic events, or FIX inhibitors were
reported. Elevated transaminases were observed in 26 out of 60
patients treated at the recommended dose and 31 out of 60 patients
received corticosteroids.
In addition to the Phase 3 trial, BEQVEZ patients have been
followed up to six years in a Phase 1/2a study and its
corresponding Phase 2a long-term follow-up study. Pfizer is
continuing to monitor for long-term treatment durability and safety
in its clinical program over the course of 15 years.
About Hemophilia B Hemophilia is a rare genetic bleeding
disorder that prevents normal blood clotting because of a
deficiency in one of several blood clotting factors and is
predominately found in males.3,4 People with hemophilia are at risk
for excessive and recurrent spontaneous and/or post-traumatic
bleeding, which can be life-threatening, particularly in those with
severe hemophilia.3,4 People with severe hemophilia often bleed
spontaneously into their muscles or joints, or rarely into other
critical closed spaces such as the intracranial space, where
bleeding can be fatal.3,4
According to the World Federation of Hemophilia, more than
38,000 people worldwide are living with hemophilia B.11 People with
hemophilia B have a deficiency in clotting FIX, a specific protein
in the blood. Hemophilia B is also called congenital FIX deficiency
or Christmas disease. The current standard of care requires
recurrent intravenous infusions of either plasma-derived or
recombinant FIX to control and prevent bleeding episodes.1,2,4
BEQVEZ (fidanacogene elaparvovec-dzkt) U.S. Important Safety
Information What is BEQVEZ? BEQVEZ is a one-time gene
therapy used for the treatment of adults with moderate to severe
hemophilia B who are receiving routine prophylaxis, have a current
life-threatening bleed or a history of life-threatening bleeds, or
have repeated serious spontaneous bleeds.
Before treatment with BEQVEZ, your healthcare professional will
conduct a blood test to check for antibodies to the AAVRh74var
virus. The results of this testing will help determine if you may
receive BEQVEZ.
Before receiving BEQVEZ, tell your healthcare professional
about all your medical conditions, including if you:
- Have kidney or liver problems, including hepatitis
- Have factor IX inhibitors or a history of factor IX
inhibitors
- Have an active infection
BEQVEZ may cause serious side effects, including:
Increased Liver Enzymes. Most patients treated with BEQVEZ
developed elevated liver enzyme levels and most did not experience
any symptoms.
Your healthcare professional will monitor liver enzymes and
factor IX activity levels before administration of BEQVEZ and
frequently following the administration to detect and identify
possible elevations in liver enzymes and to monitor your response
to BEQVEZ. Your doctor may prescribe a corticosteroid for the
treatment of elevated liver enzymes.
Avoid or limit alcohol consumption during the first year
following BEQVEZ infusion, as alcohol may reduce the effect of
BEQVEZ and may increase liver enzyme levels.
Infusion reactions, including hypersensitivity and severe
allergic reactions (anaphylaxis) may occur. Alert your healthcare
professional right away if you get any symptoms of
hypersensitivity, which may include but are not limited to low
blood pressure, fever, heart palpitation, nausea, vomiting, chills,
or headache.
BEQVEZ can insert itself into the DNA of cells in the human
body. The effect that insertion may have on those cells is unknown
but may contribute to a theoretical risk of cancer. There
have been no reported cases of cancer caused by treatment with
BEQVEZ.
The most common side effect of BEQVEZ is increased liver
enzymes. These are not all the possible side effects of BEQVEZ. For
more information, ask your healthcare professional.
Talk to your healthcare professional before receiving any
vaccinations if you are taking a corticosteroid.
Talk to your doctor about any medications you plan to take
including over the counter medications, herbal supplements, and
vitamins as certain substances can affect the liver and may
reduce the effectiveness of BEQVEZ.
Your healthcare professional will test your factor IX activity
levels and for neutralizing factor IX inhibitors.
After receiving BEQVEZ, your doctor will discuss whether and
when you are able to stop prophylaxis, if you need to resume
prophylaxis, and actions you may need to take for surgeries,
procedures, injuries, and bleeding events.
Do not donate blood, organs, tissues, or cells for
transplantation following administration of BEQVEZ.
BEQVEZ is not intended for administration to women. Males
should not donate sperm and should use a male condom or not have
sexual intercourse for up to 6 months after receiving
BEQVEZ.
Patients and caregivers should ensure proper handling of any
materials that have come into contact with the patient’s urine,
feces, saliva, mucus, or semen in the first 6 months after
BEQVEZ infusion.
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.
The full Prescribing Information can be found here. If it is not
currently available via this link, it will be visible as soon as
possible as we work to finalize the document. Please check back for
the full information shortly.
About Pfizer: Breakthroughs That Change Patients’ Lives At
Pfizer, we apply science and our global resources to bring
therapies to people that extend and significantly improve their
lives. We strive to set the standard for quality, safety and value
in the discovery, development and manufacture of health care
products, including innovative medicines and vaccines. Every day,
Pfizer colleagues work across developed and emerging markets to
advance wellness, prevention, treatments and cures that challenge
the most feared diseases of our time. Consistent with our
responsibility as one of the world's premier innovative
biopharmaceutical companies, we collaborate with health care
providers, governments and local communities to support and expand
access to reliable, affordable health care around the world. For
175 years, we have worked to make a difference for all who rely on
us. We routinely post information that may be important to
investors on our website at www.Pfizer.com. In addition, to learn
more, please visit us on www.Pfizer.com and follow us on X at
@Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook
at www.facebook.com/Pfizer/.
Category: Prescription Medicines
Disclosure notice The information contained in this
release is as of April 26, 2024. Pfizer assumes no obligation to
update forward-looking statements contained in this release as the
result of new information or future events or developments.
This release contains forward-looking information about BEQVEZ,
a gene therapy, including its potential benefits and an approval in
the U.S. of BEQVEZ for the treatment of adult patients with
hemophilia B, and Pfizer’s other investigational gene therapy and
hemophilia candidates that involves substantial risks and
uncertainties that could cause actual results to differ materially
from those expressed or implied by such statements. Risks and
uncertainties include, among other things, uncertainties regarding
the commercial success of BEQVEZ; the uncertainties inherent in
research and development, including the ability to meet anticipated
clinical endpoints, commencement and/or completion dates for our
clinical trials, regulatory submission dates, regulatory approval
dates and/or launch dates, as well as the possibility of
unfavorable new clinical data and further analyses of existing
clinical data; the risk that clinical trial data are subject to
differing interpretations and assessments by regulatory
authorities; whether regulatory authorities will be satisfied with
the design of and results from our clinical studies; whether and
when drug applications may be filed in particular jurisdictions for
BEQVEZ or any other product candidates; whether and when any
applications that may be pending or filed for BEQVEZ or any other
product candidates may be approved by regulatory authorities, which
will depend on myriad factors, including making a determination as
to whether the product's benefits outweigh its known risks and
determination of the product's efficacy and, if approved, whether
BEQVEZ or any such other product candidates will be commercially
successful; decisions by regulatory authorities impacting labeling,
manufacturing processes, safety and/or other matters that could
affect the availability or commercial potential of BEQVEZ or any
other product candidates; uncertainties regarding the impact of
COVID-19 on Pfizer’s business, operations and financial results;
and competitive developments.
A further description of risks and uncertainties can be found in
Pfizer’s Annual Report on Form 10-K for the fiscal year ended
December 31, 2023, and in its subsequent reports on Form 10-Q,
including in the sections thereof captioned “Risk Factors” and
“Forward-Looking Information and Factors That May Affect Future
Results”, as well as in its subsequent reports on Form 8-K, all of
which are filed with the U.S. Securities and Exchange Commission
and available at www.sec.gov and www.pfizer.com.
______________________________
1 Ohmori T, Mizukami H, Ozawa K, et al. New approaches to gene
and cell therapy for hemophilia. J Thromb Haemost. 2015;13(Suppl
1): S133-142.
2 Furlan R, Krishnan S, Vietri J. Patient and parent preferences
for characteristics of prophylactic treatment in hemophilia.
Patient Prefer Adherence. 2015; 9:1687-1694.
3 Mayo Clinic. Hemophilia. August 2023. Available at:
https://www.mayoclinic.org/diseases-conditions/hemophilia/symptoms-causes/syc-20373327.
Last accessed: April 2024.
4 Centers for Disease Control and Prevention. What is
hemophilia? October 2023. Available at:
https://www.cdc.gov/ncbddd/hemophilia/facts.html#:~:text=Hemophilia%20is%20usually%20an%20inherited,can%20help%20to%20stop%20bleeding.
Last accessed: April 2024.
5 Srivastava A, Santagostino E, Dougall A, et al. WFH Guidelines
for the Management of Hemophilia, 3rd edition. Haemophilia. 2020;26
(Suppl 6):1-158.
6 Burke T, Asghar S, O'Hara J, et al. Clinical, humanistic, and
economic burden of severe hemophilia B in the United States:
results from the CHESS US and CHESS US+ population surveys.
Orphanet J Rare Dis. 2021;16(1):143.
7 van Overbeeke E, Hauber B, Michelsen, et al. Patient
preferences for gene therapy in haemophilia: Results from the
PAVING threshold technique survey. Haemophilia.
2021;27(6):957-966.
8 Buckner TW, Bocharova I, Hagan K, et al. Health care resource
utilization and cost burden of hemophilia B in the United States.
Blood Adv. 2021;5(7):1954-1962.
9 Chen CX, Baker JR, Nichol MB. Economic burden of illness among
persons with hemophilia B from HUGS Vb: examining the association
of severity and treatment regimens with costs and annual bleed
rates. Value Health. 2017;20(8):1074-1082.
10 O’Hara J, Hughes D, Camp C, et al. The cost of severe
haemophilia in Europe: the CHESS study. Orphanet J Rare Dis.
2017;12(1):106.
11 World Federation of Hemophilia. Report on the annual global
survey 2021. October 2022. Available at:
https://www1.wfh.org/publications/files/pdf-2324.pdf. Last
accessed: April 2024.
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