LEUKINE (sargramostim)
Leukine (sargramostim) is a leukocyte growth factor used to accelerate myeloid reconstitution and reduce the risk of severe infections in patients undergoing chemotherapy or bone marrow transplantation. It is indicated for adult and pediatric patients following autologous or allogeneic transplants, as well as for older adults with acute myeloid leukemia after induction therapy. Additionally, the drug is used to mobilize hematopoietic progenitor cells for collection and to increase survival in patients acutely exposed to myelosuppressive doses of radiation.
How LEUKINE Works
Sargramostim is a colony-stimulating factor that binds to specific receptors on the surface of target cells to support the survival and differentiation of hematopoietic progenitor cells. It induces these cells to divide and mature into neutrophils, monocytes, macrophages, and myeloid-derived dendritic cells. The drug also activates mature granulocytes and macrophages, increasing their ability to inhibit tumor growth and respond to infections.
Development Insights
Details
- Status
- Prescription
- First Approved
- 1991-03-05
- Patent Cliff
- 2025
- Routes
- N/A
- Dosage Forms
- VIAL
LEUKINE Approval History
What LEUKINE Treats
6 indicationsLEUKINE is approved for 6 conditions since its original approval in 1991. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Acute myeloid leukemia following induction chemotherapy
- Mobilization of hematopoietic progenitor cells for autologous transplantation
- Acceleration of myeloid reconstitution following autologous bone marrow or peripheral blood progenitor cell transplantation
- Acceleration of myeloid reconstitution following allogeneic bone marrow transplantation
- Delayed neutrophil recovery or graft failure after bone marrow transplantation
- Hematopoietic Syndrome of Acute Radiation Syndrome (H-ARS)
LEUKINE Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
MoA expansion candidates
Same target(s), different indications — where else is this mechanism being explored?
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
What's emerging in LEUKINE's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications LEUKINE treats. First-in-class if their pivotal trials read out positive.
Clinical Trial Registry
74 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT07169617 | NCI-2025-06377 NCI-2025-06377, NCI24-19-01 | Ph 2 | not yet recruiting | Testing the SurVaxM Vaccine for Lung Cancer Prevention |
| NCT02339571 | NCI-2014-02674 NCI-2014-02674, EA6141 | Ph 2, Ph 3 | active not recruiting | A Phase II/III Trial of Nivolumab, Ipilimumab, and GM-CSF in Patients With Advanced Melanoma |
| NCT05455658 | RG1122380 NCI-2021-07734, UG1CA242635 | Ph 2 | active not recruiting | STEMVAC in Patients With Early Stage Triple Negative Breast Cancer |
| NCT04902703 SESAD | 19-2727 1R01AG071151-01 | Ph 2 | recruiting | Phase II Trial to Evaluate Safety and Efficacy of GM-CSF/Sargramostim in Alzheimer's Disease |
| NCT01134614 results posted | NCI-2011-02039 NCI-2011-02039, CDR0000671238 | Ph 2 | active not recruiting | Ipilimumab With or Without Sargramostim in Treating Patients With Stage III or Stage IV Melanoma That Cannot Be Removed by Surgery |
| NCT00669734 | NCI-2012-03111 NCI-2012-03111, 070602 | Ph 1 | active not recruiting | Vaccine Therapy and Sargramostim in Treating Patients With Pancreas Cancer That Cannot Be Removed By Surgery |
| NCT04237623 | NSH 1246 | Ph 2 | recruiting | GM-CSF With Post-Transplant Cyclophosphamide |
| NCT07437963 | NCI-2026-01050 NCI-2026-01050, ANBL2421 | Ph 1, Ph 2 | not yet recruiting | Testing the Addition of Iberdomide to Therapy in People With Neuroblastoma That Has Come Back, Not Responded to Treatment, or Gotten Worse |
| NCT03786783 results posted | NCI-2018-03732 NCI-2018-03732, ANBL17P1 | Ph 2 | completed | Dinutuximab, Sargramostim, and Combination Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma |
| NCT07078604 | RG1125414 NCI-2025-04327, 20925 | Ph 2 | recruiting | A Cancer Vaccine (STEMVAC) in Combination With Chemotherapy for the Treatment of PD-L1 Negative Metastatic Triple-Negative Breast Cancer |
| NCT05242965 results posted | RG1013946 NCI-2021-14159, 10726 | Ph 2 | active not recruiting | A Multiple Antigen Vaccine (STEMVAC) for the Treatment of Patients With Stage IV Non-Small Cell Lung Cancer |
| NCT02455557 results posted | I 259614 NCI-2015-00694, I 259614 | Ph 2 | active not recruiting | SurVaxM Vaccine Therapy and Temozolomide in Treating Patients With Newly Diagnosed Glioblastoma |
| NCT05269381 PNeoVCA | MC210102 NCI-2022-01258, 21-008509 | Ph 1, Ph 2 | recruiting | Personalized Neoantigen Peptide-Based Vaccine in Combination With Pembrolizumab for Treatment of Advanced Solid Tumors |
| NCT06202066 | I 3622923 NCI-2023-09345, I 3622923 | Ph 2 | recruiting | Temozolomide and Survivin Long Peptide Vaccine (SurVaxM) for the Treatment of Patients With Progressing Metastatic Neuroendocrine Carcinomas |
| NCT03126916 | ANBL1531 NCI-2016-01734, ANBL1531 | Ph 3 | recruiting | Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL) |
| NCT03794349 results posted | ANBL1821 NCI-2018-03377, ANBL1821 | Ph 2 | active not recruiting | Irinotecan Hydrochloride, Temozolomide, and Dinutuximab With or Without Eflornithine in Treating Patients With Relapsed or Refractory Neuroblastoma |
| NCT07085338 | HuNB803 NCI-2025-02707 | Ph 2 | recruiting | A Phase II Study With a Safety Run-In of the Addition of N-803 to a Chemoimmunotherapy Backbone for the Treatment of Patients With Relapsed or Refractory Neuroblastoma |
| NCT06528496 | 24-144 | Ph 2 | recruiting | N10: A Study of Reduced Chemotherapy and Monoclonal Antibody (mAb)-Based Therapy in Children With Neuroblastoma |
| NCT03879694 | I 79518 NCI-2019-00827, I 79518 | Ph 1 | completed | Survivin Long Peptide Vaccine in Treating Patients With Metastatic Neuroendocrine Tumors |
| NCT03790670 | 0839-18-FB | Ph 1 | completed | Biomarker Assessments of Leukine During Treatment of Parkinson's Disease |
| NCT05677633 | 0748-22-FB | Ph 1 | completed | Biomarker Validation Following Sargramostim Treatment in Parkinson's Disease |
| NCT04385277 results posted | ANBL19P1 NCI-2020-02950, ANBL19P1 | Ph 2 | active not recruiting | Treatment With Dinutuximab, Sargramostim (GM-CSF), and Isotretinoin in Combination With Irinotecan and Temozolomide After Intensive Therapy for People With High-Risk Neuroblastoma (NBL) |
| NCT04040231 | 17-654 | Ph 1 | completed | Using a Targeted Cancer Vaccine (Galinpepimut-S) With Immunotherapy (Nivolumab) in Mesothelioma |
| NCT04211675 STING | STUDY00000476 | Ph 1, Ph 2 | recruiting | NK Cells Infusions With Irinotecan, Temozolomide, and Dinutuximab |
| NCT02334865 | I 247913 NCI-2014-02621, I 247913 | Ph 1 | active not recruiting | SVN53-67/M57-KLH Peptide Vaccine in Treating Patients With Newly Diagnosed Multiple Myeloma Receiving Lenalidomide Maintenance Therapy |
| NCT03332667 results posted | NANT2017-01 2P01CA217959 | Ph 1 | completed | MIBG With Dinutuximab +/- Vorinostat |
| NCT04382664 results posted | UV1-202 | Ph 2 | completed | UV1 Vaccination Plus Nivolumab and Ipilimumab in Treatment of Melanoma |
| NCT02780401 WOKVAC results posted | 9626 NCI-2016-00581, UW14090 | Ph 1 | completed | Vaccine Therapy in Preventing Cancer Recurrence in Patients With Non-Metastatic, Node Positive, HER2 Negative Breast Cancer That is in Remission |
| NCT04013672 results posted | CASE6318 | Ph 2 | completed | Study of Pembrolizumab Plus SurVaxM for Glioblastoma at First Recurrence |
| NCT04642950 results posted | NPC-26-1 | Ph 2, Ph 3 | completed | A Phase II/III Study of Sargramostim |
| NCT00901511 results posted | FARM7MCPK4 Codice Interno 19900508, EudraCT 2008-007086-23 | Ph 2 | completed | Inhaled GM-CSF Therapy of Autoimmune PAP |
| NCT05101356 | UCDCC#296 NCI-2021-10667, UCDCC#296 | Ph 1, Ph 2 | recruiting | A Cancer Vaccine (Labvax 3(22)-23) and GM-CSF Alone or in Combination With Pembrolizumab for the Treatment of Advanced Stage Adenocarcinoma |
| NCT02636582 results posted | NCI-2015-02189 NCI-2015-02189, N01-CN-2012-00034 | Ph 2 | completed | Nelipepimut-S Plus GM-CSF Vaccine Therapy in Treating Patients With Breast Cancer |
| NCT02484443 results posted | NCI-2015-01001 NCI-2015-01001, AOST1421 | Ph 2 | completed | Dinutuximab in Combination With Sargramostim in Treating Patients With Recurrent Osteosarcoma |
| NCT01882010 | 0487-12-FB 2R01NS034239 | Ph 1 | completed | Leukine (Sargramostim) for Parkinson's Disease |
| NCT00803569 results posted | LUD2007-005 CDR0000628730 | Ph 1 | completed | Vaccine Therapy in Stage II, III, or IV Epithelial Ovarian, Fallopian Tube, or Primary Peritoneal Cancers |
| NCT05284214 SALIENT | PTX-001-004 | Ph 2 | withdrawn | Sargramostim With Ipilimumab Containing Therapy in Patients With Solid Tumors |
| NCT01511068 FAMPAP results posted | 2011-0959_CCHMC_IRB | Ph 2 | completed | Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP) |
| NCT04411680 iLeukPulm results posted | PTX-001-002 | Ph 2 | completed | Study of Sargramostim in Patients With COVID-19 |
| NCT02620865 results posted | 2015-100 NCI-2015-01942, 2015-100 | Ph 1, Ph 2 | completed | BATS With in Combination With Low Dose IL-1 and GM-CSF for Advanced Pancreatic Cancer |
| NCT02444546 | MC1472 NCI-2015-00665, MC1472 | Ph 1 | completed | Wild-Type Reovirus in Combination With Sargramostim in Treating Younger Patients With High-Grade Relapsed or Refractory Brain Tumors |
| NCT04707664 SCOPE results posted | PTX-001-003 | Ph 2 | completed | Sargramostim Use in COVID-19 to Recover Patient Health |
| NCT02593123 results posted | MCC-14-10739 HM20005586 | Ph 2 | completed | Adoptive Immunotherapy in Relapsed Hematological Malignancy: Early GVHD Prophylaxis |
| NCT03012100 | RU011501I NCI-2016-01878, RU011501I | Ph 2 | active not recruiting | Multi-epitope Folate Receptor Alpha Peptide Vaccine, GM-CSF, and Cyclophosphamide in Treating Patients With Triple Negative Breast Cancer |
| NCT04326920 SARPAC results posted | SARPAC | Ph 4 | completed | Sargramostim in Patients With Acute Hypoxic Respiratory Failure Due to COVID-19 (SARPAC) |
| NCT01767194 results posted | NCI-2012-03125 NCI-2012-03125, CDR0000745188 | Ph 2 | completed | Irinotecan Hydrochloride and Temozolomide With Temsirolimus or Dinutuximab in Treating Younger Patients With Refractory or Relapsed Neuroblastoma |
| NCT01700673 results posted | J1240 P01CA015396, NA_00072223 | Ph 2 | completed | Phase II Study of Azacitidine and Sargramostim as Maintenance Treatment for Poor-Risk AML or MDS |
| NCT05366283 | PTX-001-005 | Ph 1 | completed | Study to Evaluate Pharmacokinetics, Pharmacodynamics, and Safety of Sargramostim Administered Subcutaneously, Intravenously, or by Inhalation |
| NCT01673217 | I 127008 NCI-2010-00105 | Ph 1 | completed | Decitabine, Vaccine Therapy, and Pegylated Liposomal Doxorubicin Hydrochloride in Treating Patients With Recurrent Ovarian Epithelial Cancer, Fallopian Tube Cancer, or Peritoneal Cancer |
| NCT04400929 | SH-Leuk-01 | Ph 2 | completed | Using GM-CSF as a Host Directed Therapeutic Against COVID-19 |
Showing 50 of 74 trials
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
LEUKINE FDA Label Details
Indications & Usage
FDA Label (PDF)LEUKINE is indicated for the treatment of Acute myeloid leukemia following induction chemotherapy; Mobilization of hematopoietic progenitor cells for autologous transplantation; Acceleration of myeloid reconstitution following autologous bone marrow or peripheral blood progenitor cell transplantation; Acceleration of myeloid reconstitution following allogeneic bone marrow transplantation; Delayed neutrophil recovery or graft failure after bone marrow transplantation; Hematopoietic Syndrome of Acute Radiation Syndrome (H-ARS).
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment