OSAKA, Japan & CAMBRIDGE, Mass. - Friday, 31. May 2024
- 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
(BUSINESS
WIRE) -- 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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Contacts
Media Contacts:
Japanese Media
Jun Saito
jun.saito@takeda.com
U.S. and International Media
Mark Dole
mark.dole@takeda.com
