- HYMPAVZI’s approval is based on Phase 3 study results
demonstrating substantial bleed reduction compared to routine
prophylaxis and on-demand treatment in eligible patients with
hemophilia A or B without inhibitors
- In the U.S., HYMPAVZI is the first once-weekly subcutaneous
prophylactic treatment for eligible people living with hemophilia
B, and the first to be administered via a pre-filled pen or syringe
for eligible people living with hemophilia A or B
Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and
Drug Administration (FDA) has approved HYMPAVZI™ (marstacimab-hncq)
for routine prophylaxis to prevent or reduce the frequency of
bleeding episodes in adults and pediatric patients 12 years of age
and older with hemophilia A (congenital factor VIII deficiency)
without factor VIII (FVIII) inhibitors, or hemophilia B (congenital
factor IX deficiency) without factor IX (FIX) inhibitors.
HYMPAVZI is the first and only anti-tissue factor pathway
inhibitor (anti-TFPI) approved in the U.S. for the treatment of
hemophilia A or B and the first hemophilia medicine approved in the
U.S. to be administered via a pre-filled, auto-injector pen.
HYMPAVZI can offer a subcutaneous treatment option with a
once-weekly dosing schedule and minimal preparation required for
each individual administration.
“The approval of HYMPAVZI is a meaningful advancement for people
living with hemophilia A or B without inhibitors for bleed
prevention, with a generally manageable safety profile and a
straightforward once-weekly subcutaneous administration,” said
Suchitra S. Acharya, M.D., Director, Hemostasis and Thrombosis
Center Northwell Health, Program Head, Bleeding Disorders and
Thrombosis Program, Cohen Children’s Medical Center. “HYMPAVZI aims
to reduce the current treatment burden by meeting an important need
for these patients, including many who have required frequent,
time-consuming intravenous treatment infusion regimens.”
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 is Pfizer’s second hemophilia treatment to receive FDA
approval this year and is the latest meaningful scientific
advancement in our more than 40-year commitment to improve care for
people living with hemophilia,” said Aamir Malik, Chief U.S.
Commercial Officer and Executive Vice President, Pfizer. “We look
forward to launching this latest medical breakthrough and to now
offer three distinct classes of hemophilia medicines – an
anti-TFPI, gene therapy, and recombinant factor treatments – that
can meet the unique treatment needs of a wide range of
patients.”
Results from the Phase 3 BASIS trial (NCT03938792) supported the
approval of HYMPAVZI in the U.S. in adults and adolescents with
hemophilia A or B without inhibitors. In the study, HYMPAVZI
reduced the annualized bleeding rate (ABR) for treated bleeds by
35% and 92% after a 12-month active treatment period compared to
routine prophylaxis (RP) and on-demand (OD) treatment,
respectively, in patients with hemophilia A or B without
inhibitors. The safety profile for HYMPAVZI was consistent with
Phase 1/2 results. The most commonly reported adverse reactions
(≥3% of patients) in the study were injection site reactions,
headache, and pruritus.
"The hemophilia community continually seeks advancements in care
that can improve quality of life for our community members," said
Phil Gattone, President and CEO, National Bleeding Disorders
Foundation. "We greatly appreciate Pfizer's innovative efforts in
developing this novel treatment option that addresses some of the
ongoing challenges faced by people with hemophilia A and B. The
availability of this therapy represents a powerful step forward in
advancing care for more individuals and families in the bleeding
disorders community.”
The Committee for Medicinal Products for Human Use (CHMP) of the
European Medicines Agency (EMA) adopted a positive opinion for
marstacimab for the routine prophylaxis of bleeding episodes in
adults and adolescents 12 years and older with severe hemophilia A
without FVIII inhibitors, or severe hemophilia B without FIX
inhibitors. In addition to HYMPAVZI, Pfizer recently received
regulatory approvals for its hemophilia B gene therapy BEQVEZ™
(fidanacogene elaparvovec) in the U.S., EU, and Canada, and
announced positive results from a Phase 3 program investigating its
hemophilia A gene therapy (giroctocogene fitelparvovec).
About HYMPAVZI (marstacimab-hncq)
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 in the U.S. for routine prophylaxis to
prevent or reduce the frequency of bleeding episodes in adults and
pediatric patients 12 years of age and older with hemophilia A
(congenital factor VIII deficiency) without factor VIII inhibitors,
or hemophilia B (congenital factor IX deficiency) without factor IX
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 FDA approval is based on results from the BASIS study that
included 116 people living with hemophilia without inhibitors who
were treated with HYMPAVZI during a 12-month active treatment
period (ATP) versus an RP and OD intravenous regimen with FVIII or
FIX, administered as part of usual care in a six-month
observational period. During the ATP, participants received
prophylaxis (a 300 mg subcutaneous loading dose of HYMPAVZI,
followed by 150 mg subcutaneously once weekly) with potential for
dose escalation to 300 mg once weekly.
HYMPAVZI reduced the ABR for treated bleeds by 35% and 92% after
a 12-month ATP compared to RP and OD treatment, respectively, in
patients with hemophilia A or B without inhibitors. In the OD
group, superiority (p<0.0001) of HYMPAVZI was demonstrated
across all bleeding-related secondary endpoints – spontaneous
bleeds, joint bleeds, target joint bleeds, and total bleeds. In the
RP group, HYMPAVZI demonstrated non-inferiority to these secondary
efficacy endpoints.
The safety profile for HYMPAVZI was consistent with Phase 1/2
results and treatment was generally well-tolerated. The most
commonly reported adverse reactions (≥3% of patients) were
injection site reactions, headache, and pruritus.
The inhibitor cohort of the BASIS trial 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
In a survey of people in the U.S. receiving prophylaxis for
hemophilia A or B, nearly one-third of those that receive treatment
and have high compliance – defined as taking 75% or more of their
prescribed infusions – stated that the time-consuming nature of
prophylaxis was the most significant challenge of the regimen.7,8
Nearly 60% of those that took less than the prescribed number of
infusions reported that the time commitment was the primary reason
for missing infusions.
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. 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 October 11,
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, 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. 7, 24 (2012).
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. Last Reviewed:
April 2023. https://www.cdc.gov/ncbddd/hemophilia/. 6 Weyand AC,
Pipe SW. New therapies for hemophilia. Blood. 2019;133(5):389–398.
doi: https://doi.org/10.1182/blood-2018-08-872291. 7 Thornburg CD,
Duncan NA. Treatment adherence in hemophilia. Patient Prefer
Adherence. 2017;11:1677-1686. https://doi.org/10.2147/PPA.S139851.
8 Hacker MR, Geraghty S, Manco-Johnson M. Barriers to compliance
with prophylaxis therapy in haemophilia. Haemophilia.
2001;7(4):392-6.
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