FDA approves once-weekly ALTUVIIIO™, a new class
of factor VIII therapy for hemophilia A that offers significant
bleed protection
Paris and Stockholm – February
23, 2023 – The
U.S. Food and Drug Administration (FDA) has approved ALTUVIIIO™
[Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion
Protein-ehtl], previously referred to as efanesoctocog alfa, a
first-in-class, high-sustained factor VIII replacement therapy.
ALTUVIIIO is indicated for routine prophylaxis and on-demand
treatment to control bleeding episodes, as well as perioperative
management (surgery) for adults and children with hemophilia A.
ALTUVIIIO is the first and only hemophilia A treatment that
delivers normal to near-normal factor activity levels (over 40%)
for most of the week with once-weekly dosing, and significantly
reduces bleeds compared to prior factor VIII prophylaxis.
Paul HudsonCEO, Sanofi "Today’s
approval of ALTUVIIIO allows patients and physicians to reimagine
living with hemophilia. The high sustained factor activity levels
that can be achieved with ALTUVIIIO have the potential to change
the hemophilia landscape. For the first time, with a once-weekly
dose, powerful bleed protection is a reality for patients.
Significant shifts in treatment paradigms that improve people’s
lives, like ALTUVIIIO, are what we have committed to delivering at
Sanofi.”
Hemophilia A is a rare, lifelong condition in
which the ability of a person’s blood to clot properly is impaired,
leading to excessive bleeds and spontaneous bleeds into joints that
can result in joint damage and chronic pain, and potentially impact
quality of life. The severity of hemophilia is determined by the
level of clotting factor activity in a person’s blood, and there is
a negative correlation between risk of bleeding and factor activity
levels.
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“This approval marks an important clinical advancement for
the hemophilia community because we have an option that can achieve
higher levels of factor activity with a single simplified weekly
dose. By maintaining high levels of factor activity throughout the
week, patients can be confident in the bleed protection ALTUVIIIO
offers.”
This is the first approval of ALTUVIIIO. The FDA
evaluated the application under Priority Review, which is granted
to therapies that have the potential to provide significant
improvements in the treatment, diagnosis, or prevention of serious
conditions. The FDA previously granted ALTUVIIIO Breakthrough
Therapy designation in May 2022 – the first factor VIII therapy to
receive this recognition – Fast Track designation in February 2021,
and Orphan Drug designation in August 2017.
Regulatory submission in the EU is anticipated
in the second half of 2023. The European Commission granted Orphan
Drug designation in June 2019.
ALTUVIIIO helps
elevate expectations for
hemophilia A by
providing protection for
longer
The FDA approval is based on data from the
pivotal XTEND-1 Phase 3 study recently published in The New England
Journal of Medicine. Once-weekly ALTUVIIIO prophylaxis met the
primary endpoint, providing significant bleed protection for people
with severe hemophilia A with a mean annualized bleeding rate (ABR)
of 0.70 (95% CI: 0.5-1.0) and a median ABR of 0.0 (Q1, Q3: 0.0,
1.0). ALTUVIIIO met the key secondary endpoint with a significant
reduction of 77% in ABR versus prior factor prophylaxis based on an
intra-patient comparison (95% CI:58%-87%).
Additional data showed prevention of joint
bleeds with a median annualized joint bleeding rate of 0 (Q1, Q3:
0.0, 1.0). Treatment with ALTUVIIIO provided 100% resolution of
target joints, which are joints that have recurrent bleeds (e.g.,
knee, ankle, or elbow). ALTUVIIIO provided mean factor VIII
activity greater than 40% for most of the week and greater than 10%
at Day 7; these levels were associated with a low bleed risk. In
the study, ALTUVIIIO was well-tolerated and inhibitor development
to factor VIII was not detected, although is possible following
administration of ALTUVIIIO.
Additionally, interim data from XTEND-Kids
showed that children younger than 12 years of age receiving
once-weekly ALTUVIIIO for 26 weeks (n=23) experienced a mean ABR of
0.5 (95% CI: 0.2-1.3) and a median ABR of 0 (Q1, Q3: 0.0, 1.3).
Safety results were consistent with data from the XTEND-1 trial.
The full results from XTEND-Kids will be presented at a future
medical meeting.
Across the studies, ALTUVIIIO has an established
safety profile and there were no reports of factor VIII inhibitor
development, although inhibitor formation is possible following
administration of ALTUVIIIO. The most common side effects (>10%)
of ALTUVIIIO are headache and arthralgia.
ALTUVIIIO is indicated for routine prophylaxis,
on-demand treatment and control of bleeding episodes, and
perioperative management of bleeding. The simple recommended dose
of 50 IU/kg is intended for all patients and for different clinical
scenarios.
To ensure that patients have access to the
improved bleed protection provided by ALTUVIIIO, Sanofi will price
ALTUVIIIO at parity to the annual cost of treating a prophylaxis
patient on Eloctate® [Antihemophilic Factor (Recombinant), Fc
Fusion Protein]. Sanofi will also provide comprehensive patient
support services and resources online and at 1.855.MyALTUVIIIO
(855.692.5888). In the U.S., ALTUVIIIO is expected to be
commercially available in April.
About ALTUVIIIO™ALTUVIIIO [Antihemophilic Factor
(Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] is a novel von
Willebrand Factor (VWF) independent recombinant factor VIII therapy
that is designed to extend protection from bleeds with once-weekly
prophylactic dosing for adults and children with hemophilia A.
ALTUVIIIO has a 3 to 4 fold longer half-life relative to standard
and extended half-life factor VIII products. It 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.
About the XTEND Clinical ProgramsThe XTEND
clinical program is comprised of two Phase 3 trials in hemophilia
A: XTEND-1 in people 12 years or older and XTEND-Kids in children
younger than 12 years old. There is also an ongoing extension study
(XTEND-ed).
The Phase 3 XTEND-1 study
(NCT04161495) was an open-label, non-randomized
interventional study assessing the safety, efficacy, and
pharmacokinetics of once-weekly ALTUVIIIO 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 ALTUVIIIO 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 with
26 weeks of on-demand ALTUVIIIO (50 IU/kg), then switched
to once-weekly prophylaxis with ALTUVIIIO (50 IU/kg) for an
additional 26 weeks.
The primary efficacy endpoint was the
mean annualized bleeding rate (ABR) in Arm A, and the key
secondary endpoint was an intra-patient comparison of ABR during
the ALTUVIIIO weekly prophylaxis treatment period versus the
prior factor VIII prophylaxis ABR for a subset of
participants in Arm A who had participated in a
previous observational study (Study 242HA201/OBS16221).
The XTEND-Kids study (NCT04759131) was an
open-label, non-randomized interventional study of the safety,
efficacy, and pharmacokinetics of once-weekly ALTUVIIIO in
previously treated patients younger than 12 years of age (n=67)
with severe hemophilia A. Patients received once-weekly ALTUVIIIO
prophylaxis (50 IU/kg) for 52 weeks.
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 or
ALTUVIIIO™ in the US. 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 2022, revenue amounted to SEK 18.8
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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