Takeda Receives Positive CHMP Opinion for Recombinant ADAMTS13 (rADAMTS13) in Congenital Thrombotic Thrombocytopenic Purpura (cTTP)

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 TheNew 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:

1. Van Dorland H et al. Haematologica. 2019;104:2107-16
2. Joly BS et al. Blood. 2017;129(21):2836–2846
3. Scully M et al. Blood. 2017; 130:2055-63
4. Chiasakul T and Cuker A. Am Soc Hematol. 2018;2018(1):530–538
5. Alwan F et al. Blood. 2019;133:1644-51
6. Kremer Hovinga JA et al. Nat Rev Dis Primers. 2017;3:17020
7. Kremer Hovinga JA and George JN. Hereditary Thrombotic Thrombocytopenic Purpura. N Engl J Med. 2019;381(17):1653-1662
8. Orpha.net. Congenital thrombotic thrombocytopenic purpura. https://www.orpha.net/en/disease/detail/93583. Accessed May 2024
9. Zheng XL et al. J Thromb Haemost. 2020;18(10):2486-95
10. Sukumar S et al. J Clin Med. 2021;10:536

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