Press Release: NEJM publishes ALTUVIIIO XTEND-Kids phase 3 data
supporting its potential to transform the treatment landscape for
children with severe hemophilia A
NEJM publishes ALTUVIIIO XTEND-Kids phase 3
data supporting its potential to transform the treatment landscape
for children with severe hemophilia A
- ALTUVIIIO provides high-sustained
factor levels with once-weekly dosing in children under 12 with
hemophilia A
- XTEND-Kids results show highly
effective bleed protection in hemophilia A with no inhibitor
development to factor VIII
Paris, July 17, 2024 – Full
results from the XTEND-Kids phase 3 study published in The
New England Journal of Medicine (NEJM) highlights
the efficacy, safety, and pharmacokinetic profile of ALTUVIIIO
[Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein].
ALTUVIIIO (efanesoctocog alfa), a first-in-class, high-sustained
factor VIII replacement therapy, is approved for adults and
children with hemophilia A for routine prophylaxis and on-demand
treatment to control bleeding episodes as well as for perioperative
management (surgery).
Lynn Malec, MD
Medical Director of Comprehensive Center for Bleeding Disorders and
Associate Investigator at The Versiti Blood Research Institute, and
Associate Professor of Medicine and Pediatrics at The Medical
College of Wisconsin
“Children represent a population for which it has been
historically difficult to achieve effective bleed prevention and
these published results demonstrate an important breakthrough as we
strive to optimize the standard of care. Achieving high-sustained
factor activity with once-weekly dosing helps mitigate the need to
make a tradeoff between the treatment burden of factor replacement
therapy and efficacy, which we often witness in treating severe
hemophilia.”
The pivotal XTEND-Kids study published in
NEJM shows ALTUVIIIO met primary and secondary endpoints,
which included occurrence of factor VIII inhibitors and annualized
bleed rates (ABRs). The results show no inhibitor development to
factor VIII was detected with ALTUVIIIO (0% [95% confidence
interval (CI)] 0–5]). The median annualized bleed rate (ABR) was
0.00 (interquartile range [IQR]: 0.00-1.02), and the estimated mean
(95% CI) ABR was 0.61 (0.42–0.90) in the study of 73 patients
treated per protocol. In the pediatric population, clearance of
administered factor concentrates from the blood is greater than in
adults, often meaning injections are needed 2-4 times per week
using standard (SHL) or extended half-life (EHL) factor VIII
products.
Prevention of all joint bleeds is critical to
maintain joint health throughout life. Eighty-two percent of the
children treated with once-weekly ALTUVIIIO had zero joint bleeds,
demonstrating ALTUVIIIO weekly prophylaxis has the potential to
provide long-term preservation of joint health.
Dietmar Berger, MD,
PhD
Global Head of Development and Chief Medical Officer at Sanofi
“The XTEND-Kids data validate the connection between
high-sustained factor activity levels and improved health outcomes,
including joint health. Offering a treatment option that emphasizes
effective bleed protection in children with hemophilia can help
give families increased peace of mind when their loved ones
participate in everyday activities. The results are a testament to
our scientific expertise and commitment to redefine the standard of
care for children living with hemophilia through ALTUVIIIO and our
broader portfolio of hemophilia therapies.”
ALTUVIIIO was well-tolerated in children, and no
adverse events led to treatment discontinuation. The most common
treatment-emergent adverse events (>10%) were SARS-CoV-2 test
positive, upper respiratory tract infection, and fever (pyrexia).
No serious allergic reactions, anaphylaxis, or embolic or
thrombotic events were reported.
About ALTUVIIIO
ALTUVIIIO [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion
Protein] is a first-in-class high-sustained factor VIII therapy
that is designed to extend protection from bleeds with once-weekly
prophylactic dosing for adults and children with hemophilia A. In
adults and adolescents, ALTUVIIIO has a 3 to 4-fold longer
half-life relative to standard and extended half-life factor VIII
products, providing high-sustained factor activity levels within
normal to near-normal range, allowing for once-weekly
administration. ALTUVIIIO is the first factor VIII therapy that has
been shown to break through the von Willebrand factor ceiling,
which imposes a half-life limitation on earlier generation factor
VIII therapies. ALTUVIIIO builds on the innovative Fc fusion
technology by adding a region of von Willebrand factor and XTEN
polypeptides to extend its time in circulation.
ALTUVIIIO is currently approved and marketed in
the US, Taiwan, and Japan. On June 17, 2024, it was approved by the
European Commission for the treatment and prevention of bleeds and
perioperative prophylaxis in hemophilia A under the name
ALTUVOCT.
ALTUVIIIO is the first factor VIII therapy to
receive Breakthrough Therapy Designation by the US Food and
Drug Administration in May 2022, Fast Track Designation in
February 2021, and Orphan Drug Designation in 2017. The European
Commission granted orphan designation in June 2019.
About XTEND-Kids
The XTEND-Kids study (NCT04759131) was an open-label,
non-randomized interventional study of once-weekly ALTUVIIIO in
previously treated patients younger than 12 years of age with
severe hemophilia A. Patients (N=74) received once-weekly ALTUVIIIO
(50 IU/kg) prophylaxis for 52 weeks. The primary endpoint was the
occurrence of factor VIII inhibitors. Secondary endpoints included
annualized bleed rates (ABR) of treated bleeds, bleed treatment,
joint health, quality of life, perioperative management,
pharmacokinetics, and safety.
An ongoing extension study, XTEND-ed
(NCT04644575) is evaluating the long-term safety and efficacy of
ALTUVIIIO in previously treated patients with severe hemophilia A
for up to four years.
About Hemophilia A
Hemophilia A is a rare condition in which the ability of a person’s
blood to clot properly is impaired, leading to excessive and
spontaneous bleeds into joints that can result in joint damage and
chronic pain, and potentially impact quality of life. Disease
severity is determined by the level of clotting factor activity in
a person’s blood, meaning there is a negative correlation between
bleeding risk and factor activity levels.
About Sanofi and Sobi collaboration
Sobi and Sanofi collaborate on the development and
commercialization of Alprolix and Elocta/Eloctate. The
companies also collaborate on the development and commercialization
of efanesoctocog alfa, or ALTUVIIIO in the US, Taiwan, and Japan
and ALTUVOCT™ in Europe. Sobi has final development and
commercialization rights in the Sobi territory (essentially Europe,
North Africa, Russia and most Middle Eastern markets). Sanofi has
final development and commercialization rights in North America and
all other regions in the world excluding the Sobi territory.
About Sobi®
Sobi is a specialised international biopharmaceutical company
transforming the lives of people with rare and debilitating
diseases. Providing reliable access to innovative medicines in the
areas of haematology, immunology and specialty care, Sobi has
approximately 1,800 employees across Europe, North America, the
Middle East, Asia and Australia. In 2023, revenue amounted to SEK
22.1 billion. Sobi’s share (STO:SOBI) is listed on Nasdaq
Stockholm. More about Sobi at sobi.com and LinkedIn .
About Sanofi
We are an innovative global healthcare company, driven by one
purpose: we chase the miracles of science to improve people’s
lives. Our team, across the world, is dedicated to transforming the
practice of medicine by working to turn the impossible into the
possible. We provide potentially life-changing treatment options
and life-saving vaccine protection to millions of people globally,
while putting sustainability and social responsibility at the
center of our ambitions.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY
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