Press Release: FDA grants efanesoctocog alfa Breakthrough Therapy
designation for hemophilia A
FDA grants efanesoctocog alfa Breakthrough
Therapy designation for hemophilia A
- Efanesoctocog alfa is the first
factor VIII therapy to be awarded Breakthrough Therapy designation
by the FDA
- Designation is based on XTEND-1
Phase 3 study data demonstrating a clinically meaningful prevention
of bleeds and superiority in prevention of bleeding episodes
compared to prior prophylaxis factor treatment
- Efanesoctocog alfa is a novel and
investigational factor VIII therapy designed to provide normal to
near-normal factor activity levels for the majority of the week in
a once-weekly prophylactic treatment regimen
Paris and Stockholm
– June 1,
2022 – The United States Food and Drug
Administration (FDA) has granted Breakthrough Therapy designation
to efanesoctocog alfa (BIVV001) for the treatment of people with
hemophilia A, a rare and life-threatening bleeding disorder, based
on data from the pivotal XTEND-1 Phase 3 study. Sanofi and Sobi®
collaborate on the development and commercialization of
efanesoctocog alfa.
Breakthrough Therapy designation is designed to
expedite the development and review of drugs in the US that target
serious or life-threatening conditions. Drugs qualifying for this
designation must show preliminary clinical evidence that the drug
may demonstrate a substantial improvement on clinically significant
endpoints over available therapies.
John Reed, MD, PhDGlobal Head
of Research and Development at Sanofi “The Breakthrough Therapy
designation highlights efanesoctocog alfa’s potential to transform
treatment for people with hemophilia A by providing higher
protection for longer duration. This potential new class of factor
VIII therapy represents how we are boldly advancing science to
address unmet needs for the hemophilia community. We are excited to
work with regulatory authorities during the filing and review of
this innovative therapy.”
Anders Ullman, MD,
PhDHead of Research and Development and Chief Medical
Officer at Sobi“This designation supports the innovation of
efanesoctocog alfa and acknowledges its potential to fulfill an
unmet medical need for people living with hemophilia A. We are
committed to transforming lives for people living with rare
diseases, and this is a testament to the medical innovation that
science can bring.”
Topline results from the pivotal XTEND-1 Phase 3
study demonstrate efanesoctocog alfa met the primary endpoint,
showing a clinically meaningful prevention of bleeds in people with
severe hemophilia A over a 52-week period. Importantly, the key
secondary endpoint was also met, demonstrating that efanesoctocog
alfa was superior to prior prophylactic factor VIII replacement
therapy in preventing bleeding events 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.
Data from the XTEND-1 Phase 3 study are expected
to be shared at an upcoming medical meeting, and those data will
serve as the basis for submission to FDA mid-year 2022. The FDA
granted efanesoctocog alfa Orphan Drug designation in August 2017
and Fast Track designation in February 2021. The European
Commission also granted efanesoctocog alfa Orphan Drug designation
in June 2019. Regulatory submission in the EU will follow
availability of data from the ongoing XTEND-Kids pediatric study,
expected in 2023.
Hemophilia A occurs in about one in 5,000 male
births annually, and more rarely in females. It is a lifelong
condition in which the ability of a person’s blood to clot is
impaired due to a coagulation factor deficiency. People with
hemophilia can experience bleeding episodes that can cause pain,
irreversible joint damage, and life-threatening hemorrhages. Unmet
medical needs remain for people with hemophilia to strengthen
protection, reduce treatment burden, and improve quality of
life.
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, an investigational factor VIII therapy with the
potential to provide high sustained factor activity levels with
once-weekly dosing for people with hemophilia A. 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
Sanofi Contacts:Media RelationsSandrine
Guendoul | + 33 6 25 09 14 25
| sandrine.guendoul@sanofi.comSally
Bain | + 1 617 834 6026
| sally.bain@sanofi.com
Investor RelationsEva
Schaefer-Jansen | + 33 7 86 80 56 39
| eva.schaefer-jansen@sanofi.comArnaud
Delépine | + 33 6 73 69 36 93 |
arnaud.delepine@sanofi.comCorentine
Driancourt | + 33 6 40 56 92 21 |
corentine.driancourt@sanofi.comFelix
Lauscher | + 1 908 612 7239 |
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.
Investor RelationsFor details on how to contact
the Sobi Investor Relations Team, click here.
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