TTP: A Rare Blood Disorder With No Cure and a High Unmet Need

Will Sanofi and Takeda rise to the challenge?

Snigdha Gupta, Associate Director

Thrombotic thrombocytopenic purpura (TTP) has always posed a challenge for hematologists. With a mortality rate as high as 90%, if not treated early, the acute phase is characterized by the formation of blood clots that lead to organ damage and failure. Despite its deadly nature, however, TTP has been overlooked, and its low annual incidence rate of one to two cases per 100,000 individuals has hindered market growth and innovation. Only a few companies, including Sanofi and Takeda seem to recognize this untapped drug development opportunity and the urgent unmet need for innovative TTP solutions.

From Rarity to Urgency: Is the TTP Market on the Brink of Transformation?

A lack of effective treatments has so far limited TTP market growth (2020-2028: CAGR 5.3%), but with the emergence of new, innovative therapies, the market now rests at the brink of transformation. For decades, the standard TTP treatment has been plasma exchange (PEX) with high-dose corticosteroids. With over 20% of patients experiencing refractory responses and up to 40% suffering relapse, however, the need for more options is dire. As the search for more new treatments continues, adjunctive therapies like splenectomy, cyclosporine, cyclophosphamide, vincristine and rituximab have emerged as effective options when first-line therapies fail. Innovative repurposed drugs like bortezomib and N-acetylcysteine have also shown great promise, and IV immunoglobulin is gaining traction as a salvage therapy for refractory TTP.

Breaking the Mold: Potential Game Changers in TTP Treatment – Caplacizumab vs. TAK-755

TTP is a complex disease with a long list of symptoms that confound even the most seasoned physicians. It often requires a multidisciplinary approach involving hematologists, nephrologists and other specialists, which can pose logistical challenges. The first-line option of PEX is challenging and associated with weeks of hospitalization, and patients often cite¹ the trauma of “having [their] entire blood volume passed through a machine and fed back to [them] combined with strangers’ blood”. It must be clarified that TTP is not the same as idiopathic thrombocytopenic purpura (ITP). Although both blood disorders affect platelets, they have different causes and symptoms: ITP is an autoimmune disorder that results in low platelet counts (the Syk inhibitor fostamatinib² from Grifols is approved for this disease), while TTP is caused by abnormal clotting that can lead to organ damage and other serious complications.

But hope is on the horizon with two groundbreaking treatments: Caplacizumab and TAK-755. Reducing TTP-related deaths, recurrence and thromboembolic events, Sanofi’s caplacizumab is proving to be a game changer in the TTP space. Meanwhile, Takeda’s TAK-755 promises to revolutionize TTP treatment with its targeted approach that addresses the deficient ADAMTS13 enzyme upstream of the coagulation pathway. TAK-755 has shown promise in clinical trials; it reduces the time to platelet normalization, lowers the incidence of TTP-related complications and has demonstrated a 60% reduction in thrombocytopenia events compared to the standard of care (SoC). In addition to its encouraging efficacy though, TAK-755 also has lower associated incidences of adverse events compared with the SoC. Accordingly, Takeda is seeking³ marketing authorization for TAK-755 as the first recombinant ADAMTS13 replacement therapy for chronic TTP, and with its impressive safety and efficacy, lower administration frequency and improved cost-effectiveness, it is no wonder that hematologists and patients alike eagerly await its approval.

TTP Treatment Receives a Game-Changing Boost

The TTP market is poised for transformation. It cannot be ignored that caplacizumab and TAK-755 are game changers in the future TTP treatment landscape. Driven by their unique mechanisms of action and promising clinical trial results, these treatments could significantly improve outcomes and quality of life for patients with TTP. TAK-755’s safety data and projected cost-effectiveness make it an especially attractive option for patients and physicians and position it to challenge Sanofi’s caplacizumab, the current market leader in TTP treatment. Sanofi, however, is leveraging its strong brand reputation and extensive research and development capabilities to defend its position and explore new treatment options. Furthermore, mechanisms of action like BTKis¹⁰ and modalities like siRNA¹¹ will bring in novel approaches and might also prove to be disruptors in the TTP treatment landscape. But as the battle for TTP supremacy heats up, the real winners will be the patients who will benefit from the improved and innovative treatments that emerge.


  1. Answering TTP
  2. Tavalisse
  3. Takeda Announces Favorable Phase 3 Safety and Efficacy Results of TAK-755 as Compared to Standard of Care in Congenital Thrombotic Thrombocytopenic Purpura (cTTP) (January 2023)
  4. FDA approves first therapy for the treatment of adult patients with a rare blood clotting disorder,)%252C%2520a%2520rare%2520and%2520life%252D (February 2019)
  5. Study to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP) (TITAN) (June 2010)
  6. Phase III Trial With Caplacizumab in Patients With Acquired Thrombotic Thrombocytopenic Purpura (HERCULES) (September 2015)
  7. A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP) (January 2018)
  8. Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura (January 2019)
  9. Caplacizumab for Acquired Thrombotic Thrombocytopenic Purpura (February 2016)
  10. Restoring Balance in the Blood (July 2022)
  11. Personalized treatment for von Willebrand disease by RNA-targeted therapies (April 2020)
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