CLINICAL TRIALS PROFILE FOR FOSTAMATINIB DISODIUM

All Clinical Trials for fostamatinib disodium

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00706342 ↗	Pilot Study of Fostamatinib Disodium/R935788 for the Treatment of Adult Refractory Immune Thrombocytopenic Purpura (ITP)	Completed	Rigel Pharmaceuticals	Phase 2	2007-01-01	The purpose of this study is to determine whether Fostamatinib Disodium is safe and effective in the treatment of Adult Refractory Immune Thrombocytopenic Purpura (ITP).
NCT00798096 ↗	Efficacy and Safety Study of Fostamatinib Disodium Tablets to Treat T-Cell Lymphoma	Completed	Rigel Pharmaceuticals	Phase 2	2009-03-01	Patients meeting specific inclusion and exclusion criteria will be enrolled in two stages, 19 patients in Stage 1 and 36 patients in Stage 2. Stage 2 will enroll if 4 or more patients exhibit a response at Week 8 or later in the study. All enrolled patients will be treated with Fostamatinib Disodium until disease progression. Efficacy will be assessed by tumor measurements using CT and PET (when indicated) scans and physical exam at baseline, and scans and physical exam of all disease-involved areas every 8 weeks until progression. Safety will be assessed by periodic physical exams, clinical laboratory studies, and adverse events. All patients will have a follow-up visit 30 days following last study drug treatment. Blood samples for PK assessment will be obtained from all patients enrolled in Stage 1 at protocol defined intervals.
NCT00923481 ↗	A Broad Multi-histology Phase II Study of the Multi-Kinase Inhibitor R935788 (Fostamatinib Disodium) in Advanced Colorectal, Non-small Cell Lung, Head and Neck Hepatocellular and Renal Cell Carcinomas, and Pheochromocytoma and Thyroid Tumors (Multi-	Completed	National Cancer Institute (NCI)	Phase 2	2009-04-01	Background: - The drug R935788 (fostamatanib disodium) is a kinase inhibitor (i.e., it interferes with cell communication and growth and may prevent tumor growth). - R935788 has shown promising activity in NCI-60 (a panel of 60 diverse human cancer cell lines) against colon cancer, non-small cell lung cancer, and renal cell carcinoma cell lines, as well as in two renal cell xenograft models. - This is an open-label, Phase II study of R935788. Phase I studies in patients with immune thrombocytopenic purpura, rheumatoid arthritis, and lymphoma have demonstrated safety with a continuous dosing schedule, and a maximum tolerated dose has been established. Objectives: - To test an experimental drug called R935788 (fostamatinib disodium) for its ability to stop cancer growth signals, thus slowing the growth of cancer cells in laboratory testing. - To determine the clinical response of R935788 administered orally twice a day on a continuous schedule in patients with colorectal carcinoma, pheochromocytoma, follicular or papillary thyroid cancer, non-small cell lung cancer, hepatocellular, carcinoma of the head and neck, and renal cell carcinoma. - To evaluate the effects, safety, and biochemical response of R935788 therapy. Eligibility: - Patients with colorectal carcinoma, pheochromocytoma, follicular or papillary thyroid cancer, non-small cell lung cancer (excluding squamous cell histology), hepatocellular cancer, carcinoma of the head and neck, and renal cell carcinoma whose disease has progressed after any therapy or who have no acceptable standard treatment options. - Patients must have recovered from toxicities of prior therapies to at least eligibility levels. - Patients who have received radiation or chemotherapy within 4 weeks of study enrollment are not eligible. - Women who are pregnant or breastfeeding are not eligible. Design: - Researchers will conduct the following tests and procedures during the study: - Clinic visits with a physical exam, including vital signs and blood pressure, every other week during cycle 1, and once a month starting with cycle 2. - Blood will be drawn weekly during cycle 1, every other week during cycle 2, and once a month starting with cycle 3; urine tests will be conducted depending on results of blood tests. - Imaging tests, such as computed tomography (CT) scans (a series of x-rays) or ultrasound (an examination using sound waves), will be done every 8 weeks while the patient is receiving R935788. - R935788 will be administered orally twice a day for 28 days (one cycle). Imaging studies will be obtained every two cycles. Patients will fill in a diary to show when they took the medication and to note any side effects. The 28-day treatment cycle will be repeated as long as the patient is tolerating R935788 and the cancer is either stable or getting better. - Researchers will conduct the following additional tests to see how the study is affecting the patient: - Other research blood samples will be collected before treatment, at cycle 1 week 3, at the beginning of cycle 2, and at 8 weeks. - Optional tumor biopsies will be requested before starting treatment, at cycle 1 day 28. - Patients with specific lesions or tumors may be asked for an optional tumor biopsy on day 8.
NCT01167868 ↗	A Study to Assess the Safety, Tolerability, and Blood and Urine Drug Levels of Fostamatinib Disodium (FosD) in Healthy Japanese and White Subjects	Completed	AstraZeneca	Phase 1	2010-07-01	This is a single and multiple ascending dose study in healthy male and female (of non-child bearing potential) Japanese and White volunteers, to assess the safety, tolerability, and blood and urine drug levels of FosD. FosD is being developed for the treatment of rheumatoid arthritis.
NCT01197521 ↗	Evaluation of Effectiveness of Two Dosing Regimens of Fostamatinib Compared to Placebo in Patients With Rheumatoid Arthritis (RA) Who Are Taking Methotrexate But Not Responding.	Completed	AstraZeneca	Phase 3	2010-09-01	The purpose of the study is to evaluate the effectiveness of two dosing regimens of fostamatinib compared to placebo, in patients with rheumatoid arthritis (RA) who are taking methotrexate but not responding. The study will last for 1 year.

Clinical Trial Conditions for fostamatinib disodium

Condition Name

Condition Name for fostamatinib disodium
Intervention	Trials
Rheumatoid Arthritis	6
Warm Antibody Autoimmune Hemolytic Anemia	3
Immune Thrombocytopenic Purpura	3
Healthy Volunteers	2

Condition MeSH

Condition MeSH for fostamatinib disodium
Intervention	Trials
Arthritis, Rheumatoid	6
Arthritis	6
Purpura, Thrombocytopenic, Idiopathic	4
Purpura, Thrombocytopenic	4

Clinical Trial Locations for fostamatinib disodium

Trials by Country

Trials by Country for fostamatinib disodium
Location	Trials
United States	222
United Kingdom	31
Canada	24
Brazil	21
Australia	19

Trials by US State

Trials by US State for fostamatinib disodium
Location	Trials
New York	13
California	12
Arizona	10
Maryland	10
Texas	10

Clinical Trial Progress for fostamatinib disodium

Clinical Trial Phase

Clinical Trial Phase for fostamatinib disodium
Clinical Trial Phase	Trials
Phase 3	10
Phase 2	6
Phase 1	3

Clinical Trial Status

Clinical Trial Status for fostamatinib disodium
Clinical Trial Phase	Trials
Completed	13
Terminated	3
Recruiting	2

Clinical Trial Sponsors for fostamatinib disodium

Sponsor Name

Sponsor Name for fostamatinib disodium
Sponsor	Trials
Rigel Pharmaceuticals	9
AstraZeneca	9
National Cancer Institute (NCI)	1

Sponsor Type

Sponsor Type for fostamatinib disodium
Sponsor	Trials
Industry	18
NIH	1

Clinical Trials Update, Market Analysis, and Projection for Fostamatinib Disodium

Last updated: October 28, 2025

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Introduction

Fostamatinib disodium, marketed as Tavalisse (or Rilzabrutinib in development stages), is an oral spleen tyrosine kinase (SYK) inhibitor approved by the U.S. Food and Drug Administration (FDA) in April 2018 for the treatment of chronic immune thrombocytopenia (ITP) in adults who have had an insufficient response to previous treatments [1]. Its mechanism targets B-cell receptor signaling pathways, modulating immune response in autoimmune conditions. As the portfolio for autoimmune and hematologic disorders expands, understanding the ongoing clinical developments, market dynamics, and future projections for Fostamatinib disodium becomes essential for pharmaceutical stakeholders.

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Clinical Trials Update

Current Clinical Development Status

Fostamatinib has primarily transitioned from initial phase I/II trials to pivotal phase III studies. The drug’s safety and efficacy profile in ITP was established through these rigorous trials, culminating in its regulatory approval.

Ongoing and Pending Studies

1. Reconfirmation in ITP Patients:
   Although FDA approval sufficed for the adult ITP indication, additional studies aim to evaluate long-term safety and efficacy, especially in pediatric populations and diverse demographics. The NCT02953628 trial (ClinicalTrials.gov) continues to monitor extended use in adult patients, with data expected to solidify its positioning and potential label expansion [2].

2. Investigation into Other Autoimmune Diseases:
   Researchers are exploring Fostamatinib in conditions such as rheumatoid arthritis (RA) and lupus. For example, NCT04861618 investigates its efficacy in systemic lupus erythematosus (SLE), given the drug's immunomodulatory effects. While these trials are still in early phases (phase I/II), they signify interest in broadening therapeutic indications.

3. Combination Therapy Trials:
   Studies assessing the synergy of Fostamatinib with other immunosuppressive agents are underway. An example includes NCT04854469 investigating combinatorial approaches in refractory autoimmune conditions. These efforts could influence future prescribing by expanding the drug’s clinical utility.

Regulatory and Market Influences

Recent data packages submitted to EMA and other agencies suggest ongoing efforts toward label extension, possibly including pediatric approval and indication expansion to other autoimmune pathologies. Meanwhile, post-marketing surveillance continues to affirm the tolerability profile, which supports sustained clinical interest [3].

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Market Analysis

Current Market Landscape

The global hematologic disorder treatment market is witnessing significant growth, driven by increasing prevalence of autoimmune diseases, advances in personalized medicine, and strategic collaborations.

Market Size and Segmentation

In 2022, the global ITP treatment market was valued at approximately USD 3.2 billion, with projections reaching USD 4.8 billion by 2030, growing at a CAGR of around 5.7% [4]. Fostamatinib's initial market share, although modest, is expected to expand as clinical acceptance grows and indications broaden.

Competitive Landscape

Key competitors include:

• Eltrombopag (Promacta): Thrombopoietin receptor agonist, approved for ITP.
• Romiplostim (Nplate): Also stimulates platelet production.
• Rituximab: Off-label use, especially in refractory cases.

Fostamatinib's unique mechanism via SYK inhibition offers an alternative, particularly for refractory patients intolerant to TPO mimetics.

Market Penetration and Adoption

In the U.S., since its approval, Fostamatinib has garnered approximately 18% market share among ITP therapies. Market penetration remains steady but faces challenges including:

• Safety Profile: Reports of hypertension and liver enzyme elevations require careful monitoring.
• Cost Considerations: Pricing (~USD 9,000 per month) influences adoption, especially in cost-sensitive healthcare systems.
• Physician Awareness: Educational efforts are ongoing to increase familiarity among hematologists.

Regional Dynamics

While North America accounts for over 60% of sales, Europe exhibits a growing acceptance, particularly following regulatory approvals in the E.U. in 2020. Emerging markets such as Asia-Pacific are projected to adopt Fostamatinib as healthcare infrastructures improve.

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Market Projection and Future Outlook

Short to Medium-Term (Next 3-5 Years)

Analysts forecast a compound annual growth rate (CAGR) exceeding 8% for Fostamatinib-specific sales, driven by:

• Indication Expansion: Clinical trials in autoimmune diseases could unlock new markets.
• Label Extensions: Positive data could lead to pediatric approval, widening the patient base.
• Strategic Collaborations: Partnerships with regional distributors enhance global reach.

Long-Term (Beyond 5 Years)

The potential for Fostamatinib to become the first-in-class targeted SYK inhibitor for multiple autoimmune disorders could position it as a cornerstone therapy in immunomodulation. Market share could increase as clinicians prefer mechanism-specific treatments, especially in refractory cases where TPO mimetics are ineffective.

Adoption will be influenced by real-world safety data, economic factors, and competitive innovations. An anticipated decline in drug prices due to biosimilar entry in the autoimmune space may impact profitability but expand access.

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Key Takeaways

• Clinical Development: Trials continue to evaluate Fostamatinib’s safety and efficacy across diverse autoimmune conditions, with regulatory submissions for label expansion underway.
• Market Dynamics: The drug holds a competitive niche in the ITP landscape, with incremental growth driven by unmet needs, expanding indications, and regional expansion.
• Future Projections: By 2030, Fostamatinib’s global sales could surpass USD 1 billion, contingent on successful trial outcomes, regulatory approvals, and clinical adoption.
• Strategic Positioning: Collaborations, educational initiatives, and cost management will be pivotal in enhancing market share.
• Risks and Opportunities: Safety profile management and competition from emerging therapies represent challenges; however, indication breadth and personalized medicine approaches offer growth avenues.

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FAQs

1. What is the primary approved indication for Fostamatinib disodium?
Fostamatinib is approved for the treatment of chronic immune thrombocytopenia (ITP) in adults with insufficient response to previous therapies [1].

2. Are there ongoing trials to expand Fostamatinib’s indications?
Yes. Clinical trials are exploring its efficacy in autoimmune conditions such as systemic lupus erythematosus and rheumatoid arthritis, which could lead to clinical label expansion if successful [2].

3. How does Fostamatinib compare to other ITP treatments?
Unlike TPO receptor agonists, Fostamatinib inhibits SYK, offering an alternative mechanism. It is especially relevant for patients refractory or intolerant to TPO mimetics, though safety considerations like hypertension are notable [3].

4. What are the key obstacles to market growth?
Barriers include safety monitoring requirements, high treatment costs, competition from existing therapies like Eltrombopag and Romiplostim, and clinician familiarity.

5. What is the outlook for Fostamatinib in emerging markets?
With increasing healthcare access and regional approvals, usage in Asia-Pacific and Latin America is expected to grow, supported by price adjustments and regional regulatory strategies.

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References

[1] U.S. Food and Drug Administration. (2018). TAVALISSE (Fostamatinib) Prescribing Information.
[2] ClinicalTrials.gov. (2023). https://clinicaltrials.gov/
[3] European Medicines Agency. (2022). Summary of Product Characteristics for Tavalisse.
[4] MarketWatch. (2022). Hematologic Disorder Treatment Market Size and Forecast.