- In the EU, HYMPAVZI is the first once-weekly subcutaneous
treatment approved for eligible people living with severe
hemophilia B and the first to be administered via a pre-filled pen
or syringe for people living with severe hemophilia A or B
- HYMPAVZI’s approval is based on Phase 3 study results
demonstrating non-inferiority and superiority compared to routine
prophylaxis in eligible patients with hemophilia A or B without
inhibitors
Pfizer Inc. (NYSE: PFE) announced today that the European
Commission (EC) has granted marketing authorization for HYMPAVZI™
(marstacimab) for the routine prophylaxis of bleeding episodes in
patients 12 years of age and older weighing at least 35 kg with
severe hemophilia A (congenital factor VIII [FVIII] deficiency,
FVIII <1%) without FVIII inhibitors or severe hemophilia B
(congenital factor IX [FIX] deficiency, FIX <1%) without FIX
inhibitors.
HYMPAVZI is the first and only anti-tissue factor pathway
inhibitor (anti-TFPI) approved in the European Union (EU) for the
treatment of hemophilia A or B and the first hemophilia medicine
approved in the EU to be administered via a pre-filled,
auto-injector pen. HYMPAVZI offers a subcutaneous treatment option
with a once-weekly dosing schedule and minimal preparation required
for each individual administration.
“There is a considerable treatment burden associated with the
standard-of-care options for hemophilia A and B, including
time-consuming preparation and administration of infusions and
injections potentially causing missed doses and an increased risk
of bleeding,” said Dr. Laurent Frenzel, Head of the Hemophilia
Treatment and Research Center at the Necker-Enfants malades
Hospital (Paris Cité). “HYMPAVZI is a significant advancement for
eligible patients in that it may provide bleed prevention as well
as once-weekly subcutaneous administration via a pre-filled
pen.”
Hemophilia is a family of rare genetic blood diseases caused by
a clotting factor deficiency (FVIII in hemophilia A, FIX in
hemophilia B), impacting more than 800,000 people globally.1
Diagnosed in early childhood, hemophilia inhibits the blood’s
ability to clot properly, increasing the risk of repeated bleeding
inside the joints, which can lead to permanent joint damage.2,3
Despite significant progress in hemophilia treatment in recent
years, many people living with the disease continue to experience
bleeding episodes and manage their condition with frequent
intravenous infusions that may need to be administered multiple
times a week.4
“HYMPAVZI offers a first-in-class treatment option for people
living with hemophilia, a disease that often leads to recurring
joint bleeds and can impact daily activities as simple as climbing
stairs,” said Alexandre de Germay, Chief International Commercial
Officer and Executive Vice President, Pfizer. “This approval builds
on Pfizer’s more than four-decade commitment to improve the
standard of care in hemophilia, and we look forward to delivering
this medicine that reduced bleeds as compared to factor prophylaxis
and, importantly, requires limited preparation, meeting a key need
for eligible patients.”
The marketing authorization is based on results from the pivotal
Phase 3 BASIS study (NCT03938792) that evaluated the efficacy and
safety of marstacimab in adults and adolescents 12 years and older
with severe hemophilia A or B without inhibitors. In the study,
HYMPAVZI significantly reduced the annualized bleeding rate (ABR)
for treated bleeds by 35% (ABR of 5.08 vs. 7.85, p-value 0.0376)
during the 12-month active treatment period, demonstrating
non-inferiority and superiority compared to routine prophylaxis
(RP) with FVIII or FIX administered as part of usual care. The
safety profile for HYMPAVZI was consistent with Phase 1/2 results,
and the most commonly reported adverse events in the study were
injection site reactions, headache, pruritus, and hypertension.
This marketing authorization is valid in all 27 EU member
states, as well as in Iceland, Liechtenstein, and Norway. The EC
approval follows the regulatory approval of HYMPAVZI in the United
States in October.
Pfizer’s more than 40-year effort to advance hemophilia
treatment began with the introduction of recombinant treatments and
has extended to the introduction of newer, advanced treatment
modalities. In addition to recent regulatory approvals for
HYMPAVZI, Pfizer reported positive results from a Phase 3 program
investigating a gene therapy candidate in hemophilia A
(giroctocogene fitelparvovec) in July and received regulatory
approvals in Europe and the U.S. for its hemophilia B gene therapy
BEQVEZ™ (fidanacogene elaparvovec).
About HYMPAVZI (marstacimab) Discovered by Pfizer
scientists, HYMPAVZI is a rebalancing agent that targets the Kunitz
2 domain of tissue factor pathway inhibitor (TFPI), a natural
anticoagulation protein that functions to prevent the formation of
blood clots and restore hemostasis.
HYMPAVZI is approved by the EC for the routine prophylaxis of
bleeding episodes in patients aged 12 years and older weighing at
least 35 kg with severe hemophilia A (congenital factor VIII
[FVIII] deficiency, FVIII <1%) without FVIII inhibitors or
severe hemophilia B (congenital factor IX [FIX] deficiency, FIX
<1%) without FIX inhibitors.
About the BASIS study The pivotal BASIS study is a global
Phase 3, open-label, multicenter study to evaluate the efficacy and
safety of HYMPAVZI in adolescent and adult participants ages 12 to
<75 years with severe hemophilia A (defined as FVIII <1%) or
moderately severe to severe hemophilia B (defined as FIX activity
≤2%) with or without inhibitors.
The marketing authorization is based on data from 116 people
living with severe hemophilia without inhibitors who were treated
with marstacimab during a 12-month active treatment period (ATP)
versus a RP regimen with FVIII or FIX, administered as part of
usual care in a 6-month observational period. During the ATP,
participants received prophylaxis (a 300 mg subcutaneous loading
dose of marstacimab, followed by 150 mg subcutaneously once weekly)
with potential for dose escalation to 300 mg once weekly in
patients weighing ≥ 50 kg when control of bleeding events is judged
to be inadequate by the healthcare professional.
HYMPAVZI reduced the ABR for treated bleeds by 35% after a
12-month ATP compared to RP treatment in patients with hemophilia A
or B without inhibitors. In an interim analysis of the long-term
extension study, a consistent reduction in mean ABR for treated
bleeds of 2.79 (95% CI 1.90-4.09) was observed in up to an
additional 16 months of follow-up (n=87). HYMPAVZI demonstrated
non-inferiority across all bleeding-related secondary endpoints:
spontaneous bleeds, joint bleeds, target joint bleeds, and total
bleeds.
The safety profile for HYMPAVZI was consistent with Phase 1/2
results and treatment was generally well-tolerated. The most
commonly reported adverse events were injection site reactions,
headache, pruritus, and hypertension.
The inhibitor cohort of the BASIS study is ongoing, with results
expected in the third quarter of 2025. Pfizer is also conducting
BASIS KIDS, an open-label study investigating the safety and
efficacy of marstacimab in children 1 to <18 years of age with
severe hemophilia A or moderately severe to severe hemophilia B
with or without inhibitors.
About Hemophilia Hemophilia is a family of rare genetic
blood diseases caused by a clotting factor deficiency (FVIII in
hemophilia A, FIX in hemophilia B), which prevents normal blood
clotting. Hemophilia is diagnosed in early childhood and impacts
more than 800,000 people worldwide.1 The inability of the blood to
clot properly can increase the risk of painful bleeding inside the
joints, which can cause joint scarring and damage. People living
with hemophilia can suffer permanent joint damage following
repeated bleeding episodes.2,3
For decades, the most common treatment approach for hemophilia A
and B has been factor replacement therapy, which replaces the
missing clotting factors. Factor replacement therapies increase the
amount of clotting factor in the body to levels that improve
clotting, resulting in less bleeding.5,6
The burden of intravenous infusions is believed to be a barrier
to treatment adherence for some people living with hemophilia due
in part to inconvenience, time constraints, and poor venous
access.7,8,9,10 In a patient/physician/specialist nurse survey
across six European countries, lack of time for treatment and
convenience were among the leading reasons for not using the
prescribed amount of clotting factor or skipping treatment
administration.7
HYMPAVZI (marstacimab) U.S. Important Safety
Information
Important: Before you start using HYMPAVZI, it is very
important to talk to your healthcare provider about using factor
VIII and factor IX products (products that help blood clot but
work in a different way than HYMPAVZI). You may need to use factor
VIII or factor IX medicines to treat episodes of breakthrough
bleeding during treatment with HYMPAVZI. Carefully follow your
healthcare provider’s instructions regarding when to use factor
VIII or factor IX medicines and the prescribed dose during your
treatment with HYMPAVZI.
Before using HYMPAVZI, tell your healthcare provider about
all of your medical conditions, including if you:
- have a planned surgery. Your healthcare provider may stop
treatment with HYMPAVZI before your surgery. Talk to your
healthcare provider about when to stop using HYMPAVZI and when to
start it again if you have a planned surgery.
- have a severe short-term (acute) illness such as an infection
or injury.
- are pregnant or plan to become pregnant. HYMPAVZI may harm your
unborn baby. Females who are able to become pregnant:
- Your healthcare provider will do a pregnancy test before you
start your treatment with HYMPAVZI.
- You should use effective birth control (contraception) during
treatment with HYMPAVZI and for at least 2 months after the last
dose of HYMPAVZI.
- Tell your healthcare provider right away if you become pregnant
or think that you may be pregnant during treatment with
HYMPAVZI.
- are breastfeeding or plan to breastfeed. It is not known if
HYMPAVZI passes into your breast milk.
Tell your healthcare provider about all the medicines you
take, including prescription medicines, over‑the‑counter
medicines, vitamins, and herbal supplements.
What are the possible side effects of HYMPAVZI?
HYMPAVZI may cause serious side effects, including:
- blood clots (thromboembolic events). HYMPAVZI may
increase the risk for your blood to clot. Blood clots may form in
blood vessels in your arm, leg, lung, or head and can be
life‑threatening. Get medical help right away if you develop any of
these signs or symptoms of blood clots:
- swelling or pain in arms or legs
- redness or discoloration in your arms or legs
- shortness of breath
- pain in chest or upper back
- fast heart rate
- cough up blood
- feel faint
- headache
- numbness in your face
- eye pain or swelling
- trouble seeing
- allergic reactions. Allergic reactions, including rash
and itching have happened in people treated with HYMPAVZI. Stop
using HYMPAVZI and get medical help right away if you develop any
of the following symptoms of a severe allergic reaction:
- swelling of your face, lips, mouth, or tongue
- trouble breathing
- wheezing
- dizziness or fainting
- fast heartbeat or pounding in your chest
- sweating
The most common side effects of HYMPAVZI are injection site
reactions (itching, swelling, hardening, redness, bruising, pain at
the injection site), headache, and itching.
These are not all the possible side effects of HYMPAVZI. Call
your doctor for medical advice about side effects. You may report
side effects to the FDA at 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/.
Category: Prescription Medicines
Disclosure notice The information contained in this
release is as of November 20, 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
HYMPAVZI™ (marstacimab), an anti-tissue factor pathway inhibitor,
and Pfizer’s other hemophilia approved and investigational
products, including their potential benefits, 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 HYMPAVZI and
Pfizer’s other hemophilia products; 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; whether or when the inhibitor cohort of the BASIS
trial will be successful; 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 any applications may be filed with regulatory authorities in
particular jurisdictions for HYMPAVZI or any other products or
product candidates; whether and when any such applications that may
be pending or filed for HYMPAVZI or any other products or 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
HYMPAVZI or any such other products or 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 HYMPAVZI or any such other products or product candidates;
uncertainties regarding the impact of COVID-19 on our 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 World Federation of Hemophilia.
World Federation of Hemophilia Global Report on the Annual Global
Survey 2022. https://www1.wfh.org/publications/files/pdf-2399.pdf.
2 Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines
for the management of hemophilia, 3rd Edition; 2020. Haemophilia.
26(S6):1–158. https://doi.org/10.1111/hae.14046. 3 Franchini M,
Mannucci PM. Past, present and future of hemophilia: a narrative
review. Orphanet J Rare Dis. 2012;7:24.
https://doi.org/10.1186/1750-1172-7-24. 4 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. 5 Centers
for Disease Control and Prevention. Hemophilia.
https://www.cdc.gov/ncbddd/hemophilia/. 6 Weyand AC, Pipe SW. New
therapies for hemophilia. Blood. 2019;133(5):389–398.
https://doi.org/10.1182/blood-2018-08-872291. 7 De Moerloose P,
Urbancik W, Van Den Berg HM, Richards M. A survey of adherence to
haemophilia therapy in six European countries: results and
recommendations. Haemophilia. 2008;14(5):931–8. 8 Hacker MR,
Geraghty S, Manco-Johnson M. Barriers to compliance with
prophylaxis therapy in haemophilia. Haemophilia. 2001;7(4):392–6. 9
Zappa S, McDaniel M, Marandola J, Allen G. Treatment trends for
haemophilia A and haemophilia B in the United States: results from
the 2010 practice patterns survey. Haemophilia. 2012;18(3):e140–53.
10 Geraghty S, Dunkley T, Harrington C, Lindvall K, Maahs J, Sek J.
Practice patterns in haemophilia A therapy -- global progress
towards optimal care. Haemophilia. 2006;12(1):75–81.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20241120965921/en/
Media Contact: +44 (0) 1737 332 335 EUPress@pfizer.com
Investor Contact: +1 (212) 733-4848 IR@Pfizer.com
Pfizer (NYSE:PFE)
過去 株価チャート
から 11 2024 まで 12 2024
Pfizer (NYSE:PFE)
過去 株価チャート
から 12 2023 まで 12 2024
