FDA grants priority review to efanesoctocog alfa
for people with hemophilia A
- The FDA decision date for
efanesoctocog alfa, an investigational factor VIII therapy, is set
for February 28, 2023
- Priority review based on pivotal
data from the XTEND-1 Phase 3 study
- Efanesoctocog alfa delivers high
sustained factor activity levels in the normal to near-normal range
for the majority of the week with once weekly prophylaxis dosing,
providing higher protection for longer
Paris and Stockholm
– August
30, 2022 – The
U.S. Food and Drug Administration (FDA) has accepted for priority
review the Biologics License Application (BLA) for efanesoctocog
alfa (BIVV001) for the treatment of hemophilia A, a rare and
life-threatening bleeding disorder. The target action date for the
FDA decision is February 28, 2023. Sanofi and Sobi® collaborate on
the development and commercialization of efanesoctocog alfa.
Steve Pipe, MDProfessor and
Director of Pediatric Hemophilia and Coagulation Disorders Program,
University of Michigan “Factor therapy remains a cornerstone of
hemophilia treatment, but innovation has been needed in this area
to address challenges related to bleed protection and cumbersome
treatment regimens. If approved, efanesoctocog alfa can deliver
close to normal factor activity levels for the majority of the
week, potentially offering a new tier of protection. Such
therapeutic benefits would represent important advances in unmet
medical needs for people with hemophilia A and may transform the
prophylactic treatment landscape.”
The BLA is supported by data from the pivotal
XTEND-1 Phase 3 study. Results were recently presented at the 30th
International Society of Thrombosis and Haemostasis Congress. The
data demonstrate a clinically meaningful prevention of bleeds and
superiority to prior factor prophylaxis based on an intra-patient
comparison. Efanesoctocog alfa was well-tolerated, and inhibitor
development to factor VIII was not detected. The most common
treatment-emergent adverse events (>5% of participants overall)
were headache, arthralgia, fall, and back pain.
Dietmar Berger, MD, PhDGlobal
Head of Development and Chief Medical Officer at Sanofi “The
results from the pivotal XTEND-1 Phase 3 study demonstrate
efanesoctocog alfa’s ability to reduce annualized bleeding rates,
which supports its potential as a therapy with best-in-disease
efficacy. We look forward to working closely with the FDA during
the review process as we aim to bring this novel therapy to the
hemophilia A community.”
The FDA grants priority review to therapies that
have the potential to provide significant improvements in the
treatment, diagnosis, or prevention of serious conditions.
Efanesoctocog alfa received Breakthrough Therapy designation from
the FDA in May 2022 and it is the first factor VIII therapy to
receive this recognition. The FDA also granted efanesoctocog alfa
Orphan Drug designation in August 2017 and Fast Track designation
in February 2021.
Regulatory submission in the EU will follow
availability of data from the ongoing XTEND-Kids pediatric study,
with both events expected in 2023. The European Commission granted
efanesoctocog alfa Orphan Drug designation in June 2019.
About Phase 3 XTEND-1 Study (NCT04161495)The
Phase 3 XTEND-1 study (NCT04161495) was an open-label,
non-randomized interventional study assessing the safety, efficacy
and pharmacokinetics of once-weekly efanesoctocog alfa in
people 12 years of age or older (n=159) with severe hemophilia A
who were previously treated with factor VIII replacement
therapy. The study consisted of two parallel treatment arms —
the prophylaxis Arm A (n=133), in which patients who had received
prior factor VIII prophylaxis were treated with once-weekly
intravenous efanesoctocog alfa prophylaxis (50 IU/kg) for
52 weeks, and the on-demand Arm B (n=26), in which patients who had
received prior on-demand factor VIII therapy began 26
weeks of on-demand efanesoctocog alfa (50 IU/kg), then
switched to once-weekly prophylaxis (50 IU/kg) for an additional 26
weeks.
The primary efficacy endpoint was
the annualized bleeding rate (ABR) in Arm A, and the key
secondary endpoint was an intra-patient comparison of ABR during
the efanesoctocog alfa weekly prophylaxis treatment period versus
the prior factor VIII prophylaxis ABR for participants in
Arm A who had participated in a previous
observational study (Study 242HA201/OBS16221).
About hemophilia AHemophilia A is a rare,
genetic disorder in which the ability of a person’s blood to clot
is impaired due to a lack of factor VIII. Hemophilia A occurs in
about one in 5,000 male births annually, and more rarely in
females. People with hemophilia can experience bleeding episodes
that can cause pain, irreversible joint damage and life-threatening
hemorrhages. Factor replacement therapy remains a cornerstone of
care and can be used across multiple treatment scenarios.
About efanesoctocog alfa (BIVV001)Efanesoctocog
alfa is a novel and investigational recombinant factor VIII therapy
that is designed to extend protection from bleeds with once-weekly
prophylactic dosing for people with hemophilia A. It builds on the
innovative Fc fusion technology by adding a region of von
Willebrand factor and XTEN® polypeptides to extend its time in
circulation. It is the first investigational factor VIII therapy
that has been shown to break through the von Willebrand factor
ceiling, which imposes a half-life limitation on current factor
VIII therapies. Efanesoctocog alfa is currently under clinical
investigation and its safety and efficacy have not been evaluated
by any regulatory authority.
About the 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. 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 specialized international
biopharmaceutical company transforming the lives of people with
rare diseases. Providing sustainable access to innovative medicines
in the areas of haematology, immunology and specialty care, Sobi
has approximately 1,600 employees across Europe, North America, the
Middle East and Asia. In 2021, revenue amounted to SEK 15.5
billion. Sobi’s share (STO:SOBI) is listed on Nasdaq Stockholm.
More about Sobi at
sobi.com, LinkedIn and YouTube.
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 some 100
countries, 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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| sandrine.guendoul@sanofi.comSally
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| sally.bain@sanofi.comKate
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| kate.conway@sanofi.com
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| eva.schaefer-jansen@sanofi.comArnaud
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arnaud.delepine@sanofi.comCorentine
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felix.lauscher@sanofi.comPriya
Nanduri | + 1 617 764 6418 |
priya.nanduri@sanofi.com Nathalie
Pham | + 33 7 85 93 30 17 |
nathalie.pham@sanofi.com
Sobi Contacts:Media RelationsFor Sobi Media
contacts, click here.
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the Sobi Investor Relations Team, click here.
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