Takeda Receives European Commission Approval for ADZYNMA®▼ (Recombinant ADAMTS13) as the First and Only Recombinant ADAMTS13 Replacement Therapy for Congenital Thrombotic Thrombocytopenic Purpura (cTTP)
− 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
− Approval Based on Totality of Evidence, Including Results from the First Randomized, Controlled, Open-label, Crossover Phase 3 cTTP Trial
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.1,2,5 If left untreated, acute TTP events have a mortality rate of >90%.1,2
“A century after the scientific discovery of cTTP, significant unmet needs remain for patients who continue to face life-threatening acute events and debilitating chronic symptoms with limited treatment options,” said
In the Phase 3 trial, patients received 40 IU/kg ADZYNMA IV or plasma-based therapy every other week or weekly, based on regimen at enrollment for months 1-6 (period 1), crossing over to the alternate treatment for months 7-12 (period 2), and all patients received ADZYNMA for months 13-18 (period 3).3
No patient experienced an acute TTP event while receiving ADZYNMA prophylactic treatment (n=45), while there was one acute TTP event in a patient receiving plasma-based therapies (n=46).3 One subacute TTP event was reported in one patient receiving ADZYNMA during the controlled comparison periods 1 and 2, compared to seven subacute TTP events in six patients receiving plasma-based therapies.3 In the continuation phase (period 3), efficacy results – incidence rates of acute and subacute TTP events – were consistent with the results from periods 1 and 2.3
ADZYNMA demonstrated a favorable safety profile compared to plasma-based therapies. The most common adverse reactions (incidence >10%) were headache, diarrhea, dizziness, upper respiratory tract infection, nausea and migraine.3
Takeda is investigating recombinant ADAMTS13 in adults with immune-mediated thrombotic thrombocytopenic purpura (iTTP), the acquired form of TTP, in an ongoing Phase 2b trial (NCT05714969).
This approval does not result in any changes to Takeda’s consolidated forecast for the fiscal year ending
About ADZYNMA
ADZYNMA (recombinant ADAMTS13) is the first and only recombinant “A disintegrin and metalloproteinase with thrombospondin motifs 13” (ADAMTS13) enzyme replacement therapy approved for the treatment of ADAMTS13 deficiency in children and adult patients with cTTP. ADZYNMA is also approved by the
ADZYNMA was granted Orphan Drug Designation (ODD) by the
For the full list of side effects and restrictions with ADZYNMA, see the Product Information .
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.6,7 Although the exact prevalence of cTTP is unknown, estimates suggest a prevalence of 0.5-2 diagnosed cases/million.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.6 The accumulation of ultra-large VWF multimers leads to uncontrolled platelet aggregation and adhesion.5,7 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).5
cTTP has both acute and chronic manifestations (including stroke, renal and cardiovascular disease) and when left untreated, acute TTP events have a mortality rate of >90%.1,2,5 cTTP can also cause ongoing widespread organ damage and other co-morbidities resulting from an ADAMTS13-deficient state.2,7,9,10
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
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ADZYNMA is a registered trademark of
References:
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Van Dorland H et al. Haematologica. 2019;104:2107-16. - Joly BS et al. Blood. 2017;129(21):2836–2846.
- ADZYNMA (recombinant ADAMTS13) Summary of Product Characteristics; 2024.
- 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.
- 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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Japanese Media
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