Key Immune Thrombocytopenia Market Highlights
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WAYRILZ’s approval is expected to be a key immune thrombocytopenia market driver, given its first-in-class status as a reversible covalent BTK inhibitor targeting multiple immune pathways.
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Immune thrombocytopenia affects approximately 67K patients in the US, with the 7MM accounting for over 120K diagnosed prevalence cases.
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Immune thrombocytopenia gender bias: Immune thrombocytopenia is more prevalent among females as compared to males.
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Immune Thrombocytopenia Companies: Sanofi (Euronext: SAN, NASDAQ: SNY), Takeda (TSE: 4502, NYSE: TAK), Argenx (Euronext & NASDAQ: ARGX), Amgen (NASDAQ: AMGN), Novartis (NYSE: NVS), Rigel Pharmaceuticals (NASDAQ: RIGL), Sobi (NASDAQ: SOBI), Pfizer (NYSE: PFE), among others.
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The immune thrombocytopenia market size was approximately USD 1.87 billion in the US in 2023 and is projected to grow at a significant CAGR during the forecast period (2024–2034).
Immune Thrombocytopenia Market Impact and Epidemiology
According to DelveInsight’s Immune Thrombocytopenia Market Insight, Epidemiology and Market Forecast report, there is a significant unmet medical need with approximately 67K diagnosed prevalent immune thrombocytopenia cases in the US alone in 2023. Among gender-specific cases, there were nearly 21K immune thrombocytopenia diagnosed prevalence cases among males and 35K cases among females in the US.
Primary immune thrombocytopenia accounts for 80% of cases, representing idiopathic disease with no identifiable secondary cause, while secondary immune thrombocytopenia comprises 20% of cases, associated with conditions like SLE, drug reactions, infections, and other autoimmune diseases. The condition manifests differently across age groups, with adult immune thrombocytopenia often developing gradually and tending to be chronic, while childhood immune thrombocytopenia frequently resolves spontaneously, with approximately two-thirds experiencing complete remission.
Furthermore, Delveinsight report highlights that the immune thrombocytopenia treatment market is set for steady growth with robust compound annual growth rates anticipated in the coming decade. WAYRILZ’s approval is expected to be a key driver of this growth trajectory, given its first-in-class status and differentiated multi-immune modulation approach addressing underlying disease pathology.
Download the Immune Thrombocytopenia Market report to understand which other factors are driving the therapeutic market @ Immune Thrombocytopenia Market Trends.
Immune thrombocytopenia Treatment: WAYRILZ Treatment Approach
WAYRILZ represents a first-in-class oral, reversible covalent BTK inhibitor designed to target the underlying immune thrombocytopenia pathophysiology through multi-immune modulation. Unlike current treatments that primarily focus on symptom management or single pathways, WAYRILZ addresses the root cause by selectively inhibiting BTK, an enzyme critical to B-cell activation, autoantibody production, and FcγR-mediated phagocytosis.
The therapy targets both adaptive and innate immune mechanisms, preventing antibody production in B cells, blocking phagocytosis in macrophages, and demonstrating additional effects on basophils and neutrophils, while notably not affect T cells. This selective approach allows for immune thrombocytopenia modification rather than mere symptom control, offering patients a convenient oral once-daily treatment option that targets disease progression at its source.
“With its differentiated mechanism of action, Wayrilz has the potential to become a treatment of choice for immune thrombocytopenia patients who have not responded to a prior therapy,” said Brian Foard, Executive Vice President and Head of Specialty Care at Sanofi.
WAYRILZ Clinical Validation and Efficacy
The WAYRILZ FDA approval was based on compelling clinical evidence from the pivotal Phase III LUNA 3 study (NCT04562766), which demonstrated statistically significant efficacy across multiple endpoints. The randomized, multicenter trial evaluated rilzabrutinib 400 mg twice daily versus placebo in 202 adults with persistent or chronic immune thrombocytopenia.
Key WAYRILZ Clinical Trials Outcomes:
Primary Endpoint Success:
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Durable platelet response achieved in 23% of rilzabrutinib patients versus 0% on placebo (P < 0.0001)
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Overall durable response rate reached 29% when considering both double-blind and open-label periods
Secondary Endpoint Results:
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Overall platelet response: 65% with rilzabrutinib versus 33% with placebo
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52% reduction in need for rescue therapy compared to placebo (P = 0.0007)
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Significant improvement in bleeding symptoms measured by ITP Purpura Bleeding Score
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Clinically meaningful fatigue improvements observed as early as week 13, sustained through week 25
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Quality-of-life benefits observed even in patients without durable platelet response
The Phase III LUNA 3 study enrolled patients with severely refractory disease, with participants having a median of four prior ITP therapies and a median baseline platelet count of 15,000/μL (normal range: 150,000-450,000/μL). WAYRILZ was well-tolerated with a favorable safety profile, with most treatment-related adverse events being mild to moderate.
Immune Thrombocytopenia Competitive Landscape and Market Positioning
WAYRILZ enters a treatment landscape dominated by thrombopoietin receptor agonists (TPO-RAs) and traditional immunosuppressive therapies. The current immune thrombocytopenia competitive landscape includes established treatments such as PROMACTA (eltrombopag) by Novartis, which achieved the highest revenue among all medications in the 7MM in 2023, NPLATE (romiplostim) by Amgen, and DOPTELET (avatrombopag) by Sobi.
The first-line treatments typically include corticosteroids, intravenous immunoglobulin (IVIG), and anti-D immunoglobulins. Second-line options encompass TPO-RAs, splenectomy, and rituximab. Current treatments face limitations including short-duration responses, significant side effects, and limited efficacy in refractory patients.
WAYRILZ’s first-in-class BTK inhibition mechanism provides unprecedented competitive differentiation, supported by FDA Fast Track Designation and Orphan Drug Designation. The therapy addresses a critical gap for the estimated 20% of patients who don’t achieve adequate platelet responses despite receiving available first- and second-line treatments.
Explore the Immune Thrombocytopenia Drug Battle: WAYRILZ vs. other emerging therapies. Discover how these breakthrough Immune Thrombocytopenia therapies compare in efficacy, safety, cost, and market impact @ Immune Thrombocytopenia Drugs Market.
Emerging Immune Thrombocytopenia Pipeline Therapies
Several companies are actively developing next-generation immune thrombocytopenia therapies, creating a dynamic competitive environment. Takeda (TSE: 4502, NYSE: TAK) is advancing mezagitamab (TAK-079), a fully human anti-CD38 monoclonal antibody, which reported positive Phase 2 results with dose-dependent platelet improvements and up to 90.9% response rates at the highest 600mg dose.
Argenx (Euronext & NASDAQ: ARGX) achieved regulatory success in Japan with VYVGART (efgartigimod alfa), the first FcRn blocker approved for ITP, though its subcutaneous formulation failed to meet endpoints in the global ADVANCE-SC study. Pfizer (NYSE: PFE) is developing PF-06835375, a CXCR5 inhibitor, while other pipeline candidates include novel approaches targeting complement pathways and synthetic phage therapies.
The emerging pipeline also includes companies developing precision medicine approaches, combination therapies, and advanced drug delivery systems. Despite this evolving landscape, WAYRILZ’s disease-modifying mechanism and proven clinical efficacy position Sanofi to capture substantial market share in a therapeutic area with significant unmet medical needs.
Discover more Immune Thrombocytopenia pipeline therapies and the clinical development progress they are making @ Immune Thrombocytopenia Clinical Pipeline.
BTK Inhibitors: Broader WAYRILZ Therapeutic Potential
Beyond ITP, Sanofi is investigating rilzabrutinib’s potential across multiple immune-mediated diseases, leveraging BTK’s critical role in various immune cell types. The drug is currently being studied in warm autoimmune hemolytic anemia (wAIHA), IgG4-related disease (IgG4-RD), and sickle cell disease.
The European Medicines Agency has granted orphan designation for rilzabrutinib in IgG4-related disease, while regulatory reviews are ongoing in China and the European Union for the immune thrombocytopenia. This broad therapeutic potential significantly expands the addressable patient population and market opportunity beyond the initial immune thrombocytopenia approval.
Industry Expert Perspective
Clinical experts emphasize the significance of targeting underlying immune dysregulation rather than symptomatic management. “People living with immune thrombocytopenia who cannot tolerate or do not respond to medications aimed at raising platelet counts are at risk of uncontrolled bleeding and often endure side effects from steroids and other available therapies,” commented Dr. David Kuter, Director of Clinical Hematology at Massachusetts General Hospital and LUNA 3 study investigator.
“I’m encouraged by the robust therapeutic effects I’ve seen in patients of the LUNA 3 study across all aspects of the disease, including clinically meaningful and sustained improvements in platelet count, quality of life metrics, reduction in bleeding, and a favorable safety profile,” Dr. Kuter added.
Learn more about what other Industry experts are saying about WAYRILZ FDA Approval and how it will impact the Immune Thrombocytopenia treatment market @ Key Opinion Leaders on Immune Thrombocytopenia Market.
Looking Forward
The WAYRILZ approval represents a paradigm shift from symptom management to disease modification in immune thrombocytopenia treatment. DelveInsight’s analysts emphasize that over 25K US adults could benefit from this advanced treatment approach, addressing the complex immune dysregulation underlying ITP rather than merely managing its consequences.
The absence of disease-modifying therapies has highlighted a critical gap in immune thrombocytopenia treatment, with current approaches primarily focused on platelet count elevation without addressing the root pathophysiology. As the pharmaceutical industry continues to focus on precision medicine and targeted therapeutic approaches, WAYRILZ’s success demonstrates the potential for multi-immune modulation strategies to transform treatment paradigms in underserved autoimmune conditions with significant unmet medical needs.
Table of Contents
1. Key Insights
2. Executive Summary of Immune Thrombocytopenia
3. Competitive Intelligence Analysis for Immune Thrombocytopenia
4. Immune Thrombocytopenia Market Overview at a Glance
5. Immune Thrombocytopenia: Disease Background and Overview
6. Immune Thrombocytopenia Patient Journey
7. Immune Thrombocytopenia Epidemiology and Patient Population
8. Treatment Algorithm, Current Treatment, and Medical Practices
9. Immune Thrombocytopenia Unmet Needs
10. Key Endpoints of Immune Thrombocytopenia Treatment
11. Immune Thrombocytopenia Marketed Products
12. Immune Thrombocytopenia Emerging Therapies
13. Immune Thrombocytopenia: Seven Major Market Analysis
14. Attribute analysis
15. 7MM: Market Outlook
16. Access and Reimbursement Overview of Immune Thrombocytopenia
17. KOL Views
18. Immune Thrombocytopenia Market Drivers
19. Immune Thrombocytopenia Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
23. About DelveInsight
About DelveInsight
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