- A one-time dose of DURVEQTIX has reduced bleeds post-treatment
compared to standard of care with a median annualized bleed rate
(ABR) of zero bleeds (range 0 to 9.9) after up to four years of
follow-up, providing sustained bleed protection and potentially
avoiding years of treatment burden with prophylaxis for many
patients.
Pfizer Inc. (NYSE: PFE) today announced that the European
Commission (EC) has granted conditional marketing authorization for
DURVEQTIX® (fidanacogene elaparvovec), a gene therapy for the
treatment of severe and moderately severe hemophilia B (congenital
factor IX deficiency) in adult patients without a history of factor
IX inhibitors and without detectable antibodies to variant AAV
serotype Rh74. DURVEQTIX is designed to enable people living with
hemophilia B to produce factor IX (FIX) themselves via a one-time
dose, rather than multiple intravenous FIX infusions weekly or
biweekly with the current standard of care.1,2,3
“There is a substantial medical and treatment burden for people
with hemophilia B that receive standard of care today, with
frequent infusions and many remaining at risk of breakthrough
bleeds that can lead to pain and restricted mobility,” said
Alexandre de Germay, Chief International Commercial Officer and
Executive Vice President, Pfizer. “DURVEQTIX has shown the
potential to offer long-term bleed protection in a one-time dose,
reducing or eliminating bleeds for the appropriate patients with
hemophilia B. These outcomes and their impact could become
potentially transformative for hemophilia B care in the European
Union.”
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.1,4 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.1,2
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 42,000 people worldwide are living with
hemophilia B.11
The conditional marketing authorization is based on results from
the pivotal Phase 3 BENEGENE-2 study (NCT03861273) evaluating the
efficacy and safety of DURVEQTIX in adult male participants (age
18–62) with moderately severe to severe hemophilia B. BENEGENE-2
met its primary efficacy endpoint of non-inferiority and
demonstrated a statistically significant decrease in annualized
bleeding rate (ABR) for total bleeds (treated and untreated)
post-DURVEQTIX infusion versus prophylaxis regimen with FIX,
administered as part of usual care. Efficacy, based on ABR, also
remained stable during year two to year four after treatment.
DURVEQTIX was generally well-tolerated, with a safety profile
consistent with Phase 1/2 results.
This conditional marketing authorization is valid in all 27
European Union (EU) member states, as well as in Iceland,
Liechtenstein, and Norway. The EC approval follows recent
regulatory approvals by the U.S. Food and Drug Administration (FDA)
and Health Canada, where it is marketed as BEQVEZTM.
This milestone builds on Pfizer’s more than 40-year commitment
to delivering breakthrough solutions to improve the lives of people
living with hemophilia. In addition to DURVEQTIX, Pfizer recently
reported positive results from a Phase 3 program investigating a
gene therapy in hemophilia A (giroctocogene fitelparvovec).
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 for eligible patients
without inhibitors are currently under review with the FDA and
European Medicines Agency (EMA), respectively.
About DURVEQTIX® (fidanacogene elaparvovec)
DURVEQTIX is a gene therapy that contains a bio-engineered
adeno-associated virus (AAV) capsid and a high-activity variant of
human coagulation FIX gene. For people 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 needing regular
intravenous infusions of FIX, as is the current standard of
care.1,2,3
In December 2014, Pfizer licensed DURVEQTIX 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 BENEGENE-2 study is a Phase 3, open-label, single-arm study
to evaluate the efficacy and safety of DURVEQTIX 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 ABR for
participants treated with gene therapy versus FIX prophylaxis
replacement regimen, administered as part of usual care.
The study enrolled and dosed 45 participants. Eligible study
participants have completed a minimum six months of routine FIX
prophylaxis therapy during the lead-in study (NCT03587116) and
received one intravenous dose of DURVEQTIX at a dose of 5 x 1011
vg/kg. Participants in the BENEGENE-2 study were screened with a
validated assay designed to identify individuals who test negative
for neutralizing antibodies to the gene therapy vector.
The European Marketing Authorization Application was based on
the primary analysis of BENEGENE-2, which was conducted when 41
participants had reached 15 months of follow-up, with a subsequent
data cut provided during review, which monitored some patients up
to four years. Clinical trial participants will be followed for up
to a total of 15 years, including six years in the BENEGENE-2 study
and up to an additional nine years as part of a separate Phase 3
study (NCT05568719) to learn about the long-term safety and
efficacy of DURVEQTIX.
Results show that DURVEQTIX significantly reduced the frequency
of bleeding compared with usual care. At the 15-month follow-up
period, DURVEQTIX patients had a model-based ABR (total bleeds) of
1.44 compared to 4.50 during the lead-in period (p=0.0084),
resulting in a 68% reduction. DURVEQTIX eliminated bleeds in 62.2%
of patients.
DURVEQTIX was generally well-tolerated, with a safety profile
consistent with Phase 1/2 results. The most common adverse reaction
(incidence ≥5%) reported in Phase 3 and 1/2 clinical studies was an
increase in liver enzymes (transaminases), which was treated with
corticosteroids. No serious adverse events related to treatment or
associated with infusion reactions, thrombotic events, or FIX
inhibitors were reported.
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 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.
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/.
Disclosure notice
The information contained in this release is as of July 25,
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
DURVEQTIX, a gene therapy, including its potential benefits and an
approval in the EU of DURVEQTIX for the treatment of adult patients
with hemophilia B, and Pfizer’s hemophilia portfolio, 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 DURVEQTIX and
Pfizer’s hemophilia portfolio; 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 , including results from the BENEGENE-2 study and the
long-term follow-up study; 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
DURVEQTIX or any other hemophilia product candidates; whether and
when any applications that may be pending or filed for DURVEQTIX or
any other hemophilia 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 DURVEQTIX or any other
hemophilia 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 DURVEQTIX or any
other hemophilia 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 Centers for Disease
Control and Prevention. About Hemophilia. May 2024. Available from:
https://www.cdc.gov/hemophilia/about/?CDC_AAref_Val=https://www.cdc.gov/ncbddd/hemophilia/facts.html
[Last accessed: July 2024]. 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 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. 4 Mayo Clinic. Hemophilia.
August 2023. Available at:
https://www.mayoclinic.org/diseases-conditions/hemophilia/symptoms-causes/syc-20373327
[Last accessed: July 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 2022. October 2023. Available
from: https://www1.wfh.org/publications/files/pdf-2399.pdf [Last
accessed: July 2024].
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