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

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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.

Important Notice
For the purposes of this notice, “press release” means this document, any oral presentation, any question and answer session and any written or oral material discussed or distributed by Takeda Pharmaceutical Company Limited (“Takeda”) regarding this release. This press release (including any oral briefing and any question-and-answer in connection with it) is not intended to, and does not constitute, represent or form part of any offer, invitation or solicitation of any offer to purchase, otherwise acquire, subscribe for, exchange, sell or otherwise dispose of, any securities or the solicitation of any vote or approval in any jurisdiction. No shares or other securities are being offered to the public by means of this press release. No offering of securities shall be made in the United States except pursuant to registration under the U.S. Securities Act of 1933, as amended, or an exemption therefrom. This press release is being given (together with any further information which may be provided to the recipient) on the condition that it is for use by the recipient for information purposes only (and not for the evaluation of any investment, acquisition, disposal or any other transaction). Any failure to comply with these restrictions may constitute a violation of applicable securities laws.

The companies in which Takeda directly and indirectly owns investments are separate entities. In this press release, “Takeda” is sometimes used for convenience where references are made to Takeda and its subsidiaries in general. Likewise, the words “we”, “us” and “our” are also used to refer to subsidiaries in general or to those who work for them. These expressions are also used where no useful purpose is served by identifying the particular company or companies.

Forward-Looking Statements
This press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding Takeda’s future business, future position and results of operations, including estimates, forecasts, targets and plans for Takeda. Without limitation, forward-looking statements often include words such as “targets”, “plans”, “believes”, “hopes”, “continues”, “expects”, “aims”, “intends”, “ensures”, “will”, “may”, “should”, “would”, “could”, “anticipates”, “estimates”, “projects” or similar expressions or the negative thereof. These forward-looking statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those expressed or implied by the forward-looking statements: the economic circumstances surrounding Takeda’s global business, including general economic conditions in Japan and the United States; competitive pressures and developments; changes to applicable laws and regulations, including global health care reforms; challenges inherent in new product development, including uncertainty of clinical success and decisions of regulatory authorities and the timing thereof; uncertainty of commercial success for new and existing products; manufacturing difficulties or delays; fluctuations in interest and currency exchange rates; claims or concerns regarding the safety or efficacy of marketed products or product candidates; the impact of health crises, like the novel coronavirus pandemic, on Takeda and its customers and suppliers, including foreign governments in countries in which Takeda operates, or on other facets of its business; the timing and impact of post-merger integration efforts with acquired companies; the ability to divest assets that are not core to Takeda’s operations and the timing of any such 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 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.

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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Contact information

Media Contacts:
Japanese Media
Jun Saito
jun.saito@takeda.com

U.S. and International Media
Mark Dole
mark.dole@takeda.com

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