− 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
Takeda (TSE:4502/NYSE:TAK) today announced that the European
Commission (EC) approved ADZYNMA®▼ (recombinant ADAMTS13) for the
treatment of ADAMTS13 deficiency in children and adult patients
with congenital thrombotic thrombocytopenic purpura (cTTP).3
ADZYNMA is now the first and only enzyme replacement therapy in the
European Union (EU) specifically for the treatment of cTTP.3,4 This
approval includes confirmation of orphan medicinal product
designation and follows a positive opinion from the Committee for
Medicinal Products for Human Use, as announced by the company on
May 31, 2024.
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 Ricardo Marek, President,
Europe and Canada Business Unit at Takeda. “This approval marks the
first treatment specifically indicated to address the root cause of
the disease – ADAMTS13 deficiency. Building on our 70-year legacy
of innovation in rare blood disorders, we’re proud to offer ADZYNMA
to cTTP patients in the EU and remain committed to bringing
innovative medicines to rare disease patients with high unmet
need.”
The EC approval was supported by the totality of evidence
provided by the interim analysis of efficacy, pharmacokinetic,
safety and tolerability data from the first randomized, controlled
open-label, crossover Phase 3 trial in cTTP, as well as safety and
efficacy data from the continuation trial. Data from the Phase 3
trial (NCT03393975) were published in The New England Journal of
Medicine in May 2024.
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 March 31, 2025
(FY2024).
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 U.S. Food
and Drug Administration (FDA) and by the Japanese Ministry of
Health, Labour, and Welfare (MHLW) for the prophylactic and
on-demand treatment of patients with cTTP.
ADZYNMA 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 ADZYNMA. ADZYNMA was
also granted ODD by the European Medicines Agency (EMA) and
Japanese MHLW for the treatment of TTP.
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 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.
Important Notice For the purposes of this notice, “press
release” means this document, any oral presentation, any question
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Any failure to comply with these restrictions may constitute a
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with acquired companies; the ability to divest assets that are not
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divestment(s); and other factors identified in Takeda’s most recent
Annual Report on Form 20-F and Takeda’s other reports filed with
the U.S. Securities and Exchange Commission, available on Takeda’s
website at:
https://www.takeda.com/investors/sec-filings-and-security-reports/
or at https://www.sec.gov/. Takeda does not undertake to update any
of the forward-looking statements contained in this press release
or any other forward-looking statements it may make, except as
required by law or stock exchange rule. Past performance is not an
indicator of future results and the results or statements of Takeda
in this press release may not be indicative of, and are not an
estimate, forecast, guarantee or projection of Takeda’s future
results.
Medical Information This press release contains
information about products that may not be available in all
countries, or may be available under different trademarks, for
different indications, in different dosages, or in different
strengths. Nothing contained herein should be considered a
solicitation, promotion or advertisement for any prescription drugs
including the ones under development.
ADZYNMA is a registered trademark of Takeda Pharmaceuticals
International AG.
References:
- 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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version on businesswire.com: https://www.businesswire.com/news/home/20240806343138/en/
Japanese Media Jun Saito jun.saito@takeda.com
U.S. and International Media Megan Ostrower
megan.ostrower@takeda.com
Takeda Pharmaceutical (NYSE:TAK)
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