- cTTP Is an Ultra-rare, Potentially Fatal Blood-Clotting
Disorder with Limited Treatment Options; Untreated, Acute TTP
Events Have a Mortality Rate of >90%1,2
- If Approved in the European Union, rADAMTS13 Will Be the
First and Only Recombinant ADAMTS13 Enzyme Replacement Therapy for
cTTP
- Positive Opinion Based on Totality of Evidence, Including
Results from the First Randomized, Controlled, Open-label,
Crossover Phase 3 cTTP Trial
Takeda (TSE:4502/NYSE:TAK) today announced that the European
Medicines Agency’s (EMA) Committee for Medicinal Products for Human
Use (CHMP) has recommended the approval, under exceptional
circumstances, of recombinant ADAMTS13 (rADAMTS13) for the
treatment of ADAMTS13 deficiency in children and adult patients
with cTTP. The European Commission (EC) will consider the CHMP
positive opinion when determining the potential marketing
authorization for rADAMTS13 throughout the European Union (EU). If
approved, rADAMTS13 will be the first and only enzyme replacement
therapy in the EU for the treatment of cTTP.3
“People living with cTTP experience serious, potentially fatal
health challenges and have limited treatment options in the
European Union,” said Obi Umeh, M.D., M.Sc., Vice President,
Franchise Global Program Leader at Takeda. “With this positive
opinion for recombinant ADAMTS13, we are one step closer to
offering patients in the EU the first treatment specifically
indicated for cTTP. We look forward to the European Commission’s
decision as we aspire to transform the standard of care for cTTP
for more patients around the world.”
cTTP is an ultra-rare, chronic blood clotting disorder caused by
a deficiency in the ADAMTS13 enzyme.1 It is associated with acute
events and debilitating chronic symptoms or thrombotic
thrombocytopenic purpura (TTP) manifestations, which can include
thrombocytopenia, microangiopathic hemolytic anemia, renal
manifestations, stroke and abdominal pain.1,2,4 Untreated, acute
TTP events have a mortality rate of >90%.1,2
The Committee’s positive opinion was supported by the totality
of evidence including the interim analysis of efficacy,
pharmacokinetic, safety and tolerability data from the first
randomized, controlled open-label, crossover Phase 3 trial in cTTP.
Data from this trial (NCT03393975) were published in The New
England Journal of Medicine in May 2024. rADAMTS13 is also being
investigated in adults with immune-mediated thrombotic
thrombocytopenic purpura (iTTP), the acquired form of TTP, in an
ongoing Phase 2b trial (NCT05714969).
About Recombinant ADAMTS13 (rADAMTS13) rADAMTS13 is the
first and only recombinant “A disintegrin and metalloproteinase
with thrombospondin motifs 13” (ADAMTS13) enzyme replacement
therapy developed for the treatment of cTTP. Marketed in the U.S.
and Japan as ADZYNMA, rADAMTS13 was approved by the U.S. Food and
Drug Administration (FDA) and Japanese Ministry of Health, Labour
and Welfare (MHLW) for the prophylactic and on-demand treatment of
patients with cTTP.
rADAMTS13 was granted Orphan Drug Designation (ODD) by the U.S.
FDA for the treatment and prevention of TTP, including its acquired
idiopathic and secondary forms, as well as Fast Track and Rare
Pediatric Disease Designation. The U.S. FDA granted Takeda a Rare
Pediatric Disease Voucher for the approval of rADAMTS13. rADAMTS13
was also previously granted ODD by the European Medicines Agency
(EMA) and Japanese MHLW for the treatment of TTP.
About cTTP cTTP is an ultra-rare, chronic and
debilitating clotting disorder associated with life-threatening
acute events and debilitating chronic symptoms, or TTP
manifestations.5,6 TTP has an estimated prevalence of 2-6 diagnosed
cases/million. The inherited form of the disease, cTTP, accounts
for ≤5% of TTP patients.6,7,8 It develops due to deficiency in
ADAMTS13, a von Willebrand factor (VWF) cleaving protease, which
results in the accumulation of ultra-large VWF multimers in the
blood.5 The accumulation of ultra-large VWF multimers leads to
uncontrolled platelet aggregation and adhesion.4,6 This can lead to
abnormal clotting in the small blood vessels of the body and is
associated with microangiopathic hemolytic anemia and low platelet
levels (thrombocytopenia).4
cTTP has both acute and chronic manifestations (including
stroke, renal and cardiovascular disease). cTTP can also cause
ongoing widespread organ damage and other co-morbidities resulting
from an ADAMTS13-deficient state.2,6,9,10
About Takeda Takeda is focused on creating better health
for people and a brighter future for the world. We aim to discover
and deliver life-transforming treatments in our core therapeutic
and business areas, including gastrointestinal and inflammation,
rare diseases, plasma-derived therapies, oncology, neuroscience and
vaccines. Together with our partners, we aim to improve the patient
experience and advance a new frontier of treatment options through
our dynamic and diverse pipeline. As a leading values-based,
R&D-driven biopharmaceutical company headquartered in Japan, we
are guided by our commitment to patients, our people and the
planet. Our employees in approximately 80 countries and regions are
driven by our purpose and are grounded in the values that have
defined us for more than two centuries. For more information, visit
www.takeda.com.
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References:
- Van Dorland H et al. Haematologica. 2019;104:2107-16
- Joly BS et al. Blood. 2017;129(21):2836–2846
- Scully M et al. Blood. 2017; 130:2055-63
- Chiasakul T and Cuker A. Am Soc Hematol.
2018;2018(1):530–538
- Alwan F et al. Blood. 2019;133:1644-51
- Kremer Hovinga JA et al. Nat Rev Dis Primers. 2017;3:17020
- Kremer Hovinga JA and George JN. Hereditary Thrombotic
Thrombocytopenic Purpura. N Engl J Med. 2019;381(17):1653-1662
- Orpha.net. Congenital thrombotic thrombocytopenic purpura.
https://www.orpha.net/en/disease/detail/93583. Accessed May
2024
- Zheng XL et al. J Thromb Haemost. 2020;18(10):2486-95
- Sukumar S et al. J Clin Med. 2021;10:536
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version on businesswire.com: https://www.businesswire.com/news/home/20240531914145/en/
Media Contacts: Japanese Media Jun Saito
jun.saito@takeda.com
U.S. and International Media Mark Dole
mark.dole@takeda.com
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