VivoSeq
Hematology

Thrombotic Thrombocytopenic Purpura

20

Pipeline Programs↗

5

Active Trials↗

12

Companies Involved↗

Thrombotic Thrombocytopenic Purpura is an emerging landscape with 20 programs across 12 companies. The most active players are Hansa Biopharma AB, Lee's Pharmaceutical Limited, Argenx. 2 therapies are approved with 8 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

47/100

Pipeline Velocity

6/100

Mech. Concentration

29/100

Completion Rate

67/100

Attrition Rate

23/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

H

Hansa Biopharma AB

1 program

Phase 2L

Lee's Pharmaceutical Limited

1 program

Phase 2Argenx logo

Argenx

1 program

Phase 2Roche logo

Roche

1 program

Phase 2G

Genzyme, a Sanofi Company

1 program

Phase 2Takeda logo

Takeda

3 programs

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

76%

Small molecule

13 of 17 classified programs — the dominant modality here.

24%ALL OTHERSMonoclonal antibody318%Peptide16%

Small molecule leads; the ring shows all 17 classified programs, each share of the total. Modality classified for 100% of 22 pipeline programs.

Analysis

Trial Status

26

clinical trials

Completed12 · 46%
Terminated4 · 15%
Recruiting3 · 12%
Unknown status2 · 8%
Withdrawn2 · 8%
Active, not recruiting1 · 4%
Other2 · 8%

Clinical Trials

Trial Activity

26 trials

A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
Recruiting·Takeda logoTakeda·Phase 3·NCT04683003·77 enrolled
A Survey of Recombinant ADAMTS13 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
Recruiting·Takeda logoTakeda·N/A·NCT06441578·40 enrolled
A Pilot Study of Efgartigimod for Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)
Recruiting·UUniversity of Minnesota·Phase 2·NCT06831058·15 enrolled
A Study of TAK-755 (rADAMTS13) With Little to No Plasma Exchange (PEX) Treatment in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)
Active, not recruiting·Takeda logoTakeda·Phase 2·NCT05714969·33 enrolled
Treatment and Management of Patients With Congenital Thrombotic Thrombocytopenic Purpura (cTTP): An International, Multi-center Retrospective Chart Review of Patients in the Early Access Program (EAP) Treated With Recombinant ADAMTS13 (rADAMTS13)
Not yet recruiting·Takeda logoTakeda·N/A·NCT07429942·94 enrolled
Study of Cyclosporine or Corticosteroids as an Adjunct to Plasma Exchange in Thrombotic Thrombocytopenic Purpura (TTP)
Completed·OOhio State University·Phase 3·NCT00713193·26 enrolled
A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP)
Completed·BBaxalta now part of Shire·Phase 3·NCT03393975·52 enrolled
Caplacizumab and Immunosuppressive Therapy Without Firstline Therapeutic Plasma Exchange in Adults With Immune-mediated Thrombotic Thrombocytopenic Purpura
Completed·Sanofi logoSanofi·Phase 3·NCT05468320·51 enrolled
Platelet Response to Caplacizumab in the Treatment of Acquired Thrombotic Thrombocytopenic Purpura
Completed·UUniversity of Cologne·N/A·NCT05876221·223 enrolled
Study to Assess Efficacy and Safety of Anti-von Willebrand Factor (vWF) Nanobody in Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
Completed·AAblynx, a Sanofi company·Phase 2·NCT01151423·75 enrolled
Phase 1 Dose Escalation, Single Dose Study to Assess Safety and Pharmacokinetics of BAX930 in Hereditary Thrombotic Thrombocytopenic Purpura (TTP)
Completed·BBaxalta now part of Shire·Phase 1·NCT02216084·16 enrolled
Low Dose Rituximab in Thrombotic Thrombocytopenic Purpura
Completed·WWashington University School of Medicine·Phase 2·NCT01554514·19 enrolled
A Trial of Caplacizumab in Japanese Patients With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
Completed·Sanofi logoSanofi·Phase 2/3·NCT04074187·21 enrolled
Rituximab in Adult Acquired Idiopathic Thrombotic Thrombocytopenic Purpura
Completed·AAssistance Publique - Hôpitaux de Paris·Phase 2·NCT00907751·12 enrolled
The Use of Rituximab in Acute Thrombotic Thrombocytopenic Purpura (TTP)
Completed·UUniversity College, London·Phase 2·NCT00937131·40 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)