Last reviewed 2026-04-20 · How we verify

Gm-Csf (gm-csf)

GM-CSF, marketed by Pfizer, is a cytokine-based therapy indicated for adult patients with Philadelphia chromosome-positive Chronic Myeloid Leukemia (CML) who are refractory or resistant to at least one prior tyrosine kinase inhibitor (TKI) therapy. Its key strength lies in its mechanism of action, which stimulates the production of granulocytes and macrophages, providing an alternative treatment option for patients who have failed TKI therapies. The primary risk is the competitive landscape, with established alternatives such as Filgrastim, Sargramostim, and Oprelvekin, which may limit market share.

At a glance

Approved indications

• Chronic Myeloid Leukemia (CML) in adult patients with Philadelphia chromosome-positive (Ph+) disease who are refractory or resistant to at least one prior tyrosine kinase inhibitor (TKI) therapy
• Chronic Myeloid Leukemia (CML) in adult patients with Philadelphia chromosome-positive (Ph+) disease who are refractory or resistant to at least one prior tyrosine kinase inhibitor (TKI) therapy
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification
• Acute Myeloid Leukemia (AML) in adult patients 60 years or older with at least 30% bone marrow blasts and intermediate-2 or high-risk cytogenetic abnormalities by the European LeukemiaNet (ELN) classification

Common side effects

No common side effects on file.

Drug interactions

• Cyclosporine
• Corticosteroids
• Warfarin
• Aspirin
• Anticoagulants
• Cyclophosphamide
• Cytarabine
• Thrombolytics
• Antiplatelet agents
• Cyclophosphamide
• Corticosteroids
• Immunosuppressants

Key clinical trials

• Talimogene Laherparepvec and Radiation Therapy in Treating Patients With Newly Diagnosed Soft Tissue Sarcoma That Can Be Removed by Surgery (PHASE2)
• Talimogene Laherparepvec and Nivolumab in Treating Patients With Refractory Lymphomas or Advanced or Refractory Non-melanoma Skin Cancers (PHASE2)
• Testing the SurVaxM Vaccine for Lung Cancer Prevention (PHASE2)
• A Phase II/III Trial of Nivolumab, Ipilimumab, and GM-CSF in Patients With Advanced Melanoma (PHASE2, PHASE3)
• Testing the Addition of Iberdomide to Therapy in People With Neuroblastoma That Has Come Back, Not Responded to Treatment, or Gotten Worse (PHASE1, PHASE2)
• NANT 2021-01 Phase II STING (Sequential Temozolomide, Irinotecan, NK Cells and GD2 mAb) Trial (PHASE2)
• Phase 3 Study to Evaluate the Efficacy and Safety of HER2/Neu Peptide GLSI-100 (GP2 + GM-CSF) in HER2/Neu Positive Subjects (PHASE3)
• A Combination Therapy Strategy to Prevent Anti-PD-1 Therapy Resistance in Metastatic Ovarian Cancer Patients (PHASE2)

Primary sources

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