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, MDMedical 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, PhDGlobal
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 ALTUVIIIOALTUVIIIO [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-KidsThe 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 AHemophilia 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 collaborationSobi 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 SanofiWe 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
Media RelationsSandrine
Guendoul | + 33 6 25 09 14 25
| sandrine.guendoul@sanofi.comEvan Berland |
+1 215 432 0234 | evan.berland@sanofi.comNicolas
Obrist | + 33 6 77 21 27 55 |
nicolas.obrist@sanofi.comVictor
Rouault | + 33 6 70 93 71 40
| victor.rouault@sanofi.comTimothy
Gilbert | + 1 516 521 2929 |
timothy.gilbert@sanofi.com
Investor RelationsThomas Kudsk
Larsen |+ 44 7545 513 693 |
thomas.larsen@sanofi.comAlizé
Kaisserian | + 33 6 47 04 12 11 |
alize.kaisserian@sanofi.comArnaud
Delépine | + 33 6 73 69 36 93
|arnaud.delepine@sanofi.comFelix
Lauscher | + 1 908 612 7239 |
felix.lauscher@sanofi.comKeita
Browne | + 1 781 249 1766 |
keita.browne@sanofi.comNathalie Pham | +
33 7 85 93 30 17 | nathalie.pham@sanofi.comTarik
Elgoutni | + 1 617 710 3587 |
tarik.elgoutni@sanofi.comThibaud Châtelet | + 33 6
80 80 89 90 | thibaud.chatelet@sanofi.com
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StatementsThis press release contains forward-looking
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ALTUVOCT.
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