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

Rhea-AI Summary

Takeda has received a positive opinion from the EMA's CHMP for recombinant ADAMTS13 (rADAMTS13) for treating congenital thrombotic thrombocytopenic purpura (cTTP).

If approved, rADAMTS13 will be the first enzyme replacement therapy for cTTP in the EU. cTTP is an ultra-rare blood clotting disorder with a high mortality rate if untreated.

This recommendation is based on comprehensive evidence, including results from a Phase 3 trial, published in May 2024. The European Commission will now consider this opinion for potential marketing authorization in the EU.

Positive

• rADAMTS13 received a positive opinion from EMA's CHMP, a significant regulatory milestone.
• If approved, rADAMTS13 will be the first enzyme replacement therapy for cTTP in the EU.
• The recommendation is based on robust data from a Phase 3 trial, enhancing credibility.
• Potential approval could open new revenue streams in the EU market for Takeda.

Negative

• Approval is still pending from the European Commission, introducing regulatory uncertainty.
• The market for cTTP treatments is due to the ultra-rare nature of the disorder, potentially limiting revenue impact.
• The cost and time invested in clinical trials and regulatory approval processes are significant.

Insights

Medical Research Analyst

Takeda's positive CHMP opinion for recombinant ADAMTS13 (rADAMTS13) in treating congenital Thrombotic Thrombocytopenic Purpura (cTTP) is significant. This blood disorder is extremely rare and has a very high mortality rate if not treated. The lack of treatment options makes the potential approval of rADAMTS13 a groundbreaking development. It's important to note that this treatment would be the first enzyme replacement therapy for this condition in the European Union.

The recommendation is based on comprehensive evidence, including a Phase 3 clinical trial with a crossover, open-label and randomized design. These types of trials are considered robust and the publication of the data in a high-impact journal like The New England Journal of Medicine adds credibility.

Investors should understand that this development could set Takeda apart as a pioneer in the treatment of ultra-rare diseases. This not only opens up a new revenue stream but also enhances Takeda’s reputation in the medical community.

Financial Analyst

The positive CHMP opinion for rADAMTS13 has significant financial implications for Takeda. Approvals in the EU often precede approvals in other regions and this could lead to a potential increase in revenue. Considering the ultra-rare and life-threatening nature of cTTP, the drug could be priced at a premium, contributing substantially to Takeda’s top line.

Moreover, approval under 'exceptional circumstances' indicates the urgency and necessity of the treatment, which may expedite the drug’s market entry. This could result in quicker adoption and faster revenue realization.

However, investors should be aware of possible risks such as competitive pressures and the challenges of marketing a new therapy for an ultra-rare condition. Nonetheless, the overall outlook appears positive due to the unmet medical need and Takeda's established presence in the pharmaceutical industry.

• 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

OSAKA, Japan & CAMBRIDGE, Mass.--(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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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

 

View source version on businesswire.com: https://www.businesswire.com/news/home/20240531914145/en/

Media Contacts:

Japanese Media

Jun Saito

jun.saito@takeda.com

U.S. and International Media

Mark Dole

mark.dole@takeda.com

Source: Takeda Pharmaceutical Company Limited

FAQ

What is the latest update on Takeda's rADAMTS13 for cTTP?

Takeda's rADAMTS13 has received a positive opinion from the EMA's CHMP for treating congenital thrombotic thrombocytopenic purpura (cTTP).

When was the Phase 3 trial data for rADAMTS13 published?

The Phase 3 trial data for Takeda's rADAMTS13 was published in May 2024.

What is the significance of the CHMP's positive opinion for Takeda's rADAMTS13?

The CHMP's positive opinion is a key regulatory step that could lead to the approval of Takeda's rADAMTS13 as the first enzyme replacement therapy for cTTP in the EU.

What is the potential impact of rADAMTS13 approval on Takeda's stock?

If approved, rADAMTS13 could open new revenue streams in the EU, potentially positively impacting Takeda's stock price.

What are the challenges associated with Takeda's rADAMTS13 approval?

Pending approval from the European Commission and the market size due to the ultra-rare nature of cTTP are notable challenges.