CLINICAL TRIALS PROFILE FOR GILTERITINIB FUMARATE

All Clinical Trials for gilteritinib fumarate

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT04140487 ↗	Azacitidine, Venetoclax, and Gilteritinib in Treating Patients With Recurrent/Refractory FLT3-Mutated Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, or High-Risk Myelodysplastic Syndrome/Myeloproliferative Neoplasm	Recruiting	National Cancer Institute (NCI)	Phase 1/Phase 2	2019-12-17	This phase I/II trial studies the side effects and best dose of gilteritinib and to see how well it works in combination with azacitidine and venetoclax in treating patients with FLT3-mutation positive acute myeloid leukemia, chronic myelomonocytic leukemia, or high-risk myelodysplastic syndrome/myeloproliferative neoplasm that has come back (recurrent) or has not responded to treatment (refractory). Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Venetoclax may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Gilteritinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving azacitidine, venetoclax, and gilteritinib may work better compared to azacitidine and venetoclax alone in treating patients with acute myeloid leukemia, chronic myelomonocytic leukemia, or myelodysplastic syndrome/myeloproliferative neoplasm.
NCT04140487 ↗	Azacitidine, Venetoclax, and Gilteritinib in Treating Patients With Recurrent/Refractory FLT3-Mutated Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia, or High-Risk Myelodysplastic Syndrome/Myeloproliferative Neoplasm	Recruiting	M.D. Anderson Cancer Center	Phase 1/Phase 2	2019-12-17	This phase I/II trial studies the side effects and best dose of gilteritinib and to see how well it works in combination with azacitidine and venetoclax in treating patients with FLT3-mutation positive acute myeloid leukemia, chronic myelomonocytic leukemia, or high-risk myelodysplastic syndrome/myeloproliferative neoplasm that has come back (recurrent) or has not responded to treatment (refractory). Drugs used in chemotherapy, such as azacitidine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Venetoclax may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Gilteritinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving azacitidine, venetoclax, and gilteritinib may work better compared to azacitidine and venetoclax alone in treating patients with acute myeloid leukemia, chronic myelomonocytic leukemia, or myelodysplastic syndrome/myeloproliferative neoplasm.
NCT04293562 ↗	A Study to Compare Standard Chemotherapy to Therapy With CPX-351 and/or Gilteritinib for Patients With Newly Diagnosed AML With or Without FLT3 Mutations	Recruiting	National Cancer Institute (NCI)	Phase 3	2020-07-20	This phase III trial compares standard chemotherapy to therapy with CPX-351 and/or gilteritinib for patients with newly diagnosed acute myeloid leukemia with or without FLT3 mutations. Drugs used in chemotherapy, such as daunorubicin, cytarabine, and gemtuzumab ozogamicin, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. CPX-351 is made up of daunorubicin and cytarabine and is made in a way that makes the drugs stay in the bone marrow longer and could be less likely to cause heart problems than traditional anthracycline drugs, a common class of chemotherapy drug. Some acute myeloid leukemia patients have an abnormality in the structure of a gene called FLT3. Genes are pieces of DNA (molecules that carry instructions for development, functioning, growth and reproduction) inside each cell that tell the cell what to do and when to grow and divide. FLT3 plays an important role in the normal making of blood cells. This gene can have permanent changes that cause it to function abnormally by making cancer cells grow. Gilteritinib may block the abnormal function of the FLT3 gene that makes cancer cells grow. The overall goals of this study are, 1) to compare the effects, good and/or bad, of CPX-351 with daunorubicin and cytarabine on people with newly diagnosed AML to find out which is better, 2) to study the effects, good and/or bad, of adding gilteritinib to AML therapy for patients with high amounts of FLT3/ITD or other FLT3 mutations and 3) to study changes in heart function during and after treatment for AML. Giving CPX-351 and/or gilteritinib with standard chemotherapy may work better in treating patients with acute myeloid leukemia compared to standard chemotherapy alone.
NCT04293562 ↗	A Study to Compare Standard Chemotherapy to Therapy With CPX-351 and/or Gilteritinib for Patients With Newly Diagnosed AML With or Without FLT3 Mutations	Recruiting	Children's Oncology Group	Phase 3	2020-07-20	This phase III trial compares standard chemotherapy to therapy with CPX-351 and/or gilteritinib for patients with newly diagnosed acute myeloid leukemia with or without FLT3 mutations. Drugs used in chemotherapy, such as daunorubicin, cytarabine, and gemtuzumab ozogamicin, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. CPX-351 is made up of daunorubicin and cytarabine and is made in a way that makes the drugs stay in the bone marrow longer and could be less likely to cause heart problems than traditional anthracycline drugs, a common class of chemotherapy drug. Some acute myeloid leukemia patients have an abnormality in the structure of a gene called FLT3. Genes are pieces of DNA (molecules that carry instructions for development, functioning, growth and reproduction) inside each cell that tell the cell what to do and when to grow and divide. FLT3 plays an important role in the normal making of blood cells. This gene can have permanent changes that cause it to function abnormally by making cancer cells grow. Gilteritinib may block the abnormal function of the FLT3 gene that makes cancer cells grow. The overall goals of this study are, 1) to compare the effects, good and/or bad, of CPX-351 with daunorubicin and cytarabine on people with newly diagnosed AML to find out which is better, 2) to study the effects, good and/or bad, of adding gilteritinib to AML therapy for patients with high amounts of FLT3/ITD or other FLT3 mutations and 3) to study changes in heart function during and after treatment for AML. Giving CPX-351 and/or gilteritinib with standard chemotherapy may work better in treating patients with acute myeloid leukemia compared to standard chemotherapy alone.
NCT04655391 ↗	Glasdegib-Based Treatment Combinations for the Treatment of Patients With Relapsed Acute Myeloid Leukemia Who Have Undergone Hematopoietic Cell Transplantation	Not yet recruiting	National Cancer Institute (NCI)	Phase 1	2021-10-25	This phase Ib trial evaluates the best dose and effect of glasdegib in combination with venetoclax and decitabine, or gilteritinib, bosutinib, ivosidenib, or enasidenib in treating patients with acute myeloid leukemia that has come back (relapsed) after stem cell transplantation. Chemotherapy drugs, such as venetoclax and decitabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Glasdegib, bosutinib, ivosidenib, and enasidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Glasdegib inhibits the Sonic the Hedgehog gene. Venetoclax inhibits BCL-2 gene. Bosutinib is a tyrosine kinase inhibitor that inhibits BCR-ABL gene fusion. Ivosidenib inhibits isocitrate dehydrogenase-1 gene or IDH-1. Enasidenib inhibits isocitrate dehydrogenase-2 gene or IDH-2. This study involves an individualized approach that may allow doctors and researchers to more accurately predict which treatment plan works best for patients with relapsed acute myeloid leukemia.
NCT04655391 ↗	Glasdegib-Based Treatment Combinations for the Treatment of Patients With Relapsed Acute Myeloid Leukemia Who Have Undergone Hematopoietic Cell Transplantation	Not yet recruiting	City of Hope Medical Center	Phase 1	2021-10-25	This phase Ib trial evaluates the best dose and effect of glasdegib in combination with venetoclax and decitabine, or gilteritinib, bosutinib, ivosidenib, or enasidenib in treating patients with acute myeloid leukemia that has come back (relapsed) after stem cell transplantation. Chemotherapy drugs, such as venetoclax and decitabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Glasdegib, bosutinib, ivosidenib, and enasidenib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Glasdegib inhibits the Sonic the Hedgehog gene. Venetoclax inhibits BCL-2 gene. Bosutinib is a tyrosine kinase inhibitor that inhibits BCR-ABL gene fusion. Ivosidenib inhibits isocitrate dehydrogenase-1 gene or IDH-1. Enasidenib inhibits isocitrate dehydrogenase-2 gene or IDH-2. This study involves an individualized approach that may allow doctors and researchers to more accurately predict which treatment plan works best for patients with relapsed acute myeloid leukemia.

Clinical Trial Conditions for gilteritinib fumarate

Condition Name

Condition Name for gilteritinib fumarate
Intervention	Trials
Recurrent Acute Myeloid Leukemia	2
Recurrent High Risk Myelodysplastic Syndrome	1
Recurrent Myelodysplastic/Myeloproliferative Neoplasm	1
Refractory Acute Myeloid Leukemia	1

Condition MeSH

Condition MeSH for gilteritinib fumarate
Intervention	Trials
Leukemia, Myeloid, Acute	2
Leukemia, Myeloid	2
Leukemia	2
Preleukemia	1

Clinical Trial Locations for gilteritinib fumarate

Trials by Country

Trials by Country for gilteritinib fumarate
Location	Trials
United States	48
Canada	6
Puerto Rico	1

Trials by US State

Trials by US State for gilteritinib fumarate
Location	Trials
California	2
Texas	2
Maryland	1
Maine	1
Louisiana	1

Clinical Trial Progress for gilteritinib fumarate

Clinical Trial Phase

Clinical Trial Phase for gilteritinib fumarate
Clinical Trial Phase	Trials
Phase 3	1
Phase 1/Phase 2	1
Phase 1	1

Clinical Trial Status

Clinical Trial Status for gilteritinib fumarate
Clinical Trial Phase	Trials
Recruiting	2
Not yet recruiting	1

Clinical Trial Sponsors for gilteritinib fumarate

Sponsor Name

Sponsor Name for gilteritinib fumarate
Sponsor	Trials
National Cancer Institute (NCI)	3
M.D. Anderson Cancer Center	1
Children's Oncology Group	1

Sponsor Type

Sponsor Type for gilteritinib fumarate
Sponsor	Trials
NIH	3
Other	3

Clinical Trials Update, Market Analysis, and Projection for Gilteritinib Fumarate

Last updated: November 3, 2025

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Introduction

Gilteritinib fumarate, marketed as Xospata, is a targeted therapy developed by Astellas Pharma for the treatment of relapsed or refractory acute myeloid leukemia (AML) harboring FLT3 mutations. Since its FDA approval in 2018, gilteritinib has gained prominence within hematologic oncology, owing to its mechanism as a potent FLT3 inhibitor. This report synthesizes recent clinical trial activities, market dynamics, and projections to inform stakeholders’ strategic decisions.

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Clinical Trials Update

Regulatory Approvals and Ongoing Trials

Following its FDA approval in November 2018 for FLT3-mutated AML, gilteritinib has been subjected to numerous clinical investigations to expand its therapeutic scope. The pivotal ADMIRAL trial (NCT02421939) established its efficacy and safety profile in relapsed/refractory AML, demonstrating a superior overall survival (OS) compared to standard chemotherapy (median OS: 9.3 vs. 5.6 months).

Post-approval, the drug's clinical pipeline includes several studies:

• Combination Therapy Trials: Multiple Phase 1 and 2 studies assess gilteritinib combined with chemotherapeutics or immunotherapies to enhance remission rates.
• Frontline Settings: Trials like NCT04070461 evaluate gilteritinib as a first-line agent in newly diagnosed FLT3-mutated AML patients, aiming to broaden its usage.
• Expanding Indications: Investigations into gilteritinib's efficacy for other hematologic malignancies, such as myelodysplastic syndromes (MDS), are underway, although results are preliminary.

Recent Data and Updates

In 2022, Astellas announced updated survival data from ongoing follow-up studies reinforcing gilteritinib's durability of response (DoR) and manageable safety profile. Notably:

• The median DoR extended beyond 16 months in some patient cohorts.
• Resistance mechanisms, including secondary mutations like F691L, continue to be explored, guiding next-generation FLT3 inhibitor development.
• Real-world evidence from registries indicates improved survival metrics comparable to clinical trial outcomes, broadening confidence in its real-world applicability.

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Market Analysis

Market Landscape and Competitive Positioning

Gilteritinib's initial success established it as a primary FLT3 inhibitor in AML, competing with agents like midostaurin (Rydapt) and quizartinib (under regulatory review). Key market factors include:

• Market Penetration: As of 2023, gilteritinib maintains a dominant position in the relapsed/refractory AML niche, with estimated global sales reaching approximately $350 million.
• Pricing and Reimbursement: The average wholesale price (AWP) per 28-day cycle ranges from $25,000 to $30,000, influenced by regional healthcare policies. Reimbursement strategies are evolving, with payer adoption driven by demonstrated clinical benefit and cost-effectiveness.

Geographical Expansion and Geographic Markets

• United States: The primary market with extensive utilization, supported by streamlined regulatory pathways.
• Europe: Regulatory approval obtained in the European Union, with commercialization expanding across major markets like Germany, France, and the UK.
• Asia: Growth potential exists, especially in Japan and China, where AML prevalence is high. Regulatory submissions are ongoing or planned, with localized clinical data supporting approval.

Market Drivers and Challenges

• Drivers:

  • Rising incidence of AML among aging populations.
  • Advancements in molecular diagnostics facilitating FLT3 mutation testing.
  • Clinical trial results favoring gilteritinib's efficacy over traditional chemotherapies.

• Challenges:

  • Resistance mutations limiting long-term efficacy.
  • Competition from emerging FLT3 inhibitors with potentially superior profiles.
  • High treatment costs affecting reimbursement and access.

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Market Projection and Future Outlook

Sales Forecast

Based on current trends, market research indicates that gilteritinib's global sales are poised to grow at a compound annual growth rate (CAGR) of 12-15% over the next five years, driven by:

• Expansion into frontline treatment protocols.
• Increased adoption in Asia-Pacific markets.
• Improved diagnostic penetration enabling targeted therapy.

By 2028, projected revenues could reach $800 million to $1 billion, assuming successful approval for additional indications and combination regimens.

Key Growth Opportunities

• Indication Expansion: Demonstrating efficacy in upfront AML and other hematologic malignancies.
• Combination Regimens: Synergistic use with hypomethylating agents or immune checkpoint inhibitors to achieve deeper and more durable responses.
• Biomarker Development: Enhanced precision medicine guided by molecular profiling, increasing patient stratification and therapy optimization.

Competitive Dynamics and R&D Outlook

Emerging FLT3 inhibitors like FF-1048 and tandem inhibitors aim to overcome resistance issues. As such, gilteritinib's market share might face pressure unless continuous innovation occurs—through label expansions, personalized medicine integration, or next-generation formulations.

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Key Takeaways

• Clinical validation of gilteritinib’s efficacy continues through ongoing trials, with promising data supporting its use in new therapeutic contexts.
• The market remains sizable, with sustained growth potential attributed to expanding indications and regional penetration.
• Pricing strategies and payer reimbursement will heavily influence future sales trajectories.
• The competitive landscape necessitates ongoing innovation, including combination therapies and resistance management.
• Strategic partnerships and investments in biomarker research** will be critical for maximizing market share and therapeutic impact.

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FAQs

1. What is the current regulatory status of gilteritinib globally?
Gilteritinib is FDA-approved in the United States and has obtained approval or CE marking in Europe. Regulatory submissions are ongoing or planned in Asia, notably in Japan and China, for broader access.

2. How does gilteritinib compare to other FLT3 inhibitors?
Gilteritinib offers potent inhibitory activity against FLT3 mutations, including internal tandem duplication (ITD) and tyrosine kinase domain (TKD) mutations. It demonstrates a favorable safety profile, but emerging agents with higher selectivity or resistance mitigation strategies are under development.

3. What are the main resistance mechanisms associated with gilteritinib?
Secondary mutations such as F691L reduce drug binding affinity, leading to resistance. Ongoing research aims to identify and target these mutations or develop next-generation inhibitors.

4. What are the prospects for gilteritinib in first-line AML therapy?
Initial trials suggest potential for gilteritinib as a standalone frontline agent, especially in patients unfit for intensive chemotherapy. Results from ongoing Phase 3 studies will clarify its role in this setting.

5. How might pricing and reimbursement strategies evolve?
Cost-effectiveness analyses and real-world outcome data will influence pricing negotiations. Payers may favor combination regimens that improve outcomes, potentially impacting reimbursement models favorably for gilteritinib.

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References

1. FDA. (2018). FDA approves gilteritinib for relapsed or refractory AML with FLT3 mutations.
2. Astellas Pharma. (2022). Gilteritinib (Xospata) Clinical Data Update.
3. Market Research Future. (2023). Global AML Therapeutics Market Analysis and Forecast.
4. ClinicalTrials.gov. (Accessed 2023).
5. European Medicines Agency. (2022). Gilteritinib approval status.