DelveInsight’s “Immune Thrombocytopenia – Pipeline Insight, 2025” explores a dynamic and diversified therapeutic landscape with over 30 emerging assets targeting different facets of the immune system. The next wave of ITP therapies focuses on novel immunomodulatory mechanisms such as FcRn inhibition, spleen tyrosine kinase (Syk) blockade, B-cell depletion, BTK inhibition, and complement pathway regulation—ushering in a new era of targeted and steroid-sparing interventions.
Key pharmaceutical companies such as UCB Pharma, Rigel Pharmaceuticals, Sanofi, Jiangsu HengRui Medicine, Amgen, and Novartis are leading efforts to redefine the treatment paradigm. Rozanolixizumab, UCB’s FcRn inhibitor, has demonstrated encouraging results in reducing pathogenic IgG levels and improving platelet counts in chronic ITP patients. Similarly, Sanofi’s rilzabrutinib, an oral BTK inhibitor, is gaining attention for its favorable safety profile and potential to offer durable responses in both newly diagnosed and relapsed patients.
Fostamatinib, a Syk inhibitor from Rigel Pharmaceuticals, is already approved in the U.S. and is being evaluated for expanded indications and earlier lines of therapy. Meanwhile, advanced trials are also underway for agents such as efgartigimod and sutimlimab, reflecting the growing trend toward mechanism-specific treatments that modulate key drivers of autoimmunity without broadly suppressing the immune system.
DelveInsight’s report provides a deep dive into the mechanisms of action, clinical development timelines, regulatory pathways, and competitive landscape shaping the future of ITP therapy. As precision immunology continues to advance, the ITP treatment landscape is transitioning from generalized immune suppression to refined, targeted therapies that offer long-term disease control with improved safety and patient adherence.
Interested in learning more about the current treatment landscape and the key drivers shaping the immune thrombocytopenia pipeline? Dive in now!
Key Takeaways from the Immune Thrombocytopenia Pipeline Report
• DelveInsight’s immune thrombocytopenia pipeline analysis depicts a strong space with 30+ active players working to develop 30+ pipeline drugs for immune thrombocytopenia treatment.
• The leading immune thrombocytopenia companies include UCB, arGEN-X, HanAll Biopharma/Immunovant, Momenta Pharmaceuticals, Principia Biopharma, Hutchison MediPharma, Cour Pharmaceutical Development, and others, which are evaluating their lead assets to improve the immune thrombocytopenia treatment landscape.
• Key immune thrombocytopenia pipeline therapies in various stages of development include Rozanolixizumab (UCB7665), Efgartigimod, Batoclimab, M 254, Rilzabrutinib, HMPL 523, CNP-105, and others.
• In February 2025, Novartis' Ianalumab, for the treatment of primary immune thrombocytopenia, was granted FDA orphan designation, according to a post on the agency’s website.
• In May 2024, CASI Pharmaceuticals (Nasdaq: CASI) announced that after submitting an IND application for CID-103 on April 12, the FDA approved the company to proceed with a Phase 1/2 study of CID-103 in adults with chronic Immune Thrombocytopenia (ITP) as of May 13.
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Immune Thrombocytopenia Overview
Immune Thrombocytopenia (ITP) is an autoimmune disorder characterized by a low platelet count caused by the immune system mistakenly attacking and destroying platelets, which are essential for blood clotting. This can lead to easy bruising, bleeding, and prolonged bleeding times. ITP can be acute or chronic and affects both children and adults.
Diagnosis is typically made by excluding other causes of thrombocytopenia and confirming low platelet levels through blood tests. Treatment varies depending on severity and may include corticosteroids, immunoglobulins, thrombopoietin receptor agonists, or immunosuppressive therapies to increase platelet counts and reduce bleeding risks.
Immune Thrombocytopenia Treatment Analysis: Drug Profile
Rozanolixizumab (UCB7665): UCB
Rozanolixizumab is a humanized, high-affinity monoclonal antibody (IgG4P) targeting the neonatal Fc receptor (FcRn). By inhibiting IgG recycling, this subcutaneously administered antibody aims to reduce pathogenic autoantibody levels and improve outcomes in IgG-mediated autoimmune diseases. It has received orphan drug designation for immune thrombocytopenia (ITP) from both the FDA (April 2018) and the European Commission (January 2019). Rozanolixizumab is currently in Phase III trials for idiopathic thrombocytopenic purpura and myasthenia gravis, as well as Phase II trials for chronic inflammatory demyelinating polyradiculoneuropathy and encephalitis.
Efgartigimod: arGEN-X
Efgartigimod is a first-in-class investigational antibody fragment designed to target the neonatal Fc receptor (FcRn). It is being developed to treat severe autoimmune diseases driven by pathogenic IgG autoantibodies, addressing significant unmet medical needs. Efgartigimod is in the registration phase for myasthenia gravis and is undergoing Phase III clinical trials for primary immune thrombocytopenia (ITP), chronic inflammatory demyelinating polyradiculoneuropathy, and pemphigus vulgaris.
Discover the emerging therapies transforming immune thrombocytopenia treatment here: https://www.delveinsight.com/sample-request/immune-thrombocytopenia-itp-pipeline-insight
Immune Thrombocytopenia Therapeutics Assessment
By Product Type
• Mono
• Combination
• Mono/Combination.
By Stage
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
By Route of Administration
• Oral
• Parenteral
• Intravitreal
• Subretinal
• Topical
By Molecule Type
• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy
Scope of the Immune Thrombocytopenia Pipeline Report
• Coverage: Global
• Key Immune Thrombocytopenia Companies: UCB, arGEN-X, HanAll Biopharma/Immunovant, Momenta Pharmaceuticals, Principia Biopharma, Hutchison MediPharma, Cour Pharmaceutical Development, and others.
• Key Immune Thrombocytopenia Pipeline Therapies: Rozanolixizumab (UCB7665), Efgartigimod, Batoclimab, M 254, Rilzabrutinib, HMPL 523, CNP-105, and others.
Take a deep dive into key insights on leading and emerging therapies for Immune Thrombocytopenia!
Table of Contents
1. Introduction
2. Executive Summary
3. Immune Thrombocytopenia Pipeline: Overview
4. Analytical Perspective In-depth Commercial Assessment
5. Immune Thrombocytopenia Pipeline Therapeutics
6. Immune Thrombocytopenia Pipeline: Late-Stage Products (Phase III)
7. Immune Thrombocytopenia Pipeline: Mid-Stage Products (Phase II)
8. Immune Thrombocytopenia Pipeline: Early Stage Products (Phase I)
9. Therapeutic Assessment
10. Inactive Products
11. Company-University Collaborations (Licensing/Partnering) Analysis
12. Key Companies
13. Key Products
14. Unmet Needs
15. Market Drivers and Barriers
16. Future Perspectives and Conclusion
17. Analyst Views
18. Appendix
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