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:
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Van Dorland H et al. Haematologica. 2019;104:2107-16
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Joly BS et al. Blood. 2017;129(21):2836–2846
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Scully M et al. Blood. 2017; 130:2055-63
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Chiasakul T and Cuker A. Am Soc Hematol. 2018;2018(1):530–538
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Alwan F et al. Blood. 2019;133:1644-51
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Kremer Hovinga JA et al. Nat Rev Dis Primers. 2017;3:17020
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Kremer Hovinga JA and George JN. Hereditary Thrombotic Thrombocytopenic Purpura. N Engl J Med. 2019;381(17):1653-1662
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Orpha.net. Congenital thrombotic thrombocytopenic purpura. https://www.orpha.net/en/disease/detail/93583. Accessed May 2024
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Zheng XL et al. J Thromb Haemost. 2020;18(10):2486-95
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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
