LOMUSTINE
Lomustine is an alkylating drug used for patients with brain tumors, including those that are primary or have spread from other parts of the body. It is typically administered after a patient has already undergone surgical or radiation procedures. This medication also helps patients with Hodgkin’s lymphoma when used alongside other chemotherapies after the disease has progressed following initial chemotherapy.
How LOMUSTINE Works
This medication works by alkylating DNA and RNA within cells. It also interferes with several key enzymatic processes by attaching to amino acids in proteins through a process called carbamoylation.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2025-10-27
- Routes
- ORAL
- Dosage Forms
- CAPSULE
LOMUSTINE Approval History
What LOMUSTINE Treats
2 indicationsLOMUSTINE is approved for 2 conditions since its original approval in 2025. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Brain Tumor
- Hodgkin's Lymphoma
LOMUSTINE Boxed Warning
DELAYED MYELOSUPPRESSION and RISK OF OVERDOSAGE DELAYED MYELOSUPPRESSION Lomustine Capsules cause myelosuppression including fatal myelosuppression. Myelosuppression is delayed, dose-related, and cumulative; occurring 4 to 6 weeks after drug administration and persisting for 1 to 2 weeks. Thrombocytopenia is generally more severe than leukopenia. Cumulative myelosuppression from Lomustine Capsules is manifested by greater severity and longer duration of cytopenias. Monitor blood counts for at le...
WARNING: DELAYED MYELOSUPPRESSION and RISK OF OVERDOSAGE DELAYED MYELOSUPPRESSION Lomustine Capsules cause myelosuppression including fatal myelosuppression. Myelosuppression is delayed, dose-related, and cumulative; occurring 4 to 6 weeks after drug administration and persisting for 1 to 2 weeks. Thrombocytopenia is generally more severe than leukopenia. Cumulative myelosuppression from Lomustine Capsules is manifested by greater severity and longer duration of cytopenias. Monitor blood counts for at least 6 weeks after each dose. Do not give Lomustine Capsules more frequently than every 6 weeks [see Warnings and Precautions (5.1) , Dosage and Administration (2.2 , 2.3) ]. RISK OF OVERDOSAGE PRESCRIBE, DISPENSE, AND ADMINISTER ONLY ENOUGH CAPSULES FOR ONE DOSE. Fatal toxicity occurs with overdosage of Lomustine Capsules. Both physician and pharmacist should emphasize to the patient that only one dose of Lomustine Capsules is taken every 6 weeks [see Dosage and Administration (2.1) , Warnings and Precautions (5.2) , Overdosage (10) ]. WARNING: DELAYED MYELOSUPPRESSION and RISK OF OVERDOSAGE See full prescribing information for complete boxed warning. Delayed Myelosuppression Lomustine Capsules cause myelosuppression including fatal myelosuppression. Myelosuppression is delayed, dose-related, and cumulative. Thrombocytopenia is generally more severe than leukopenia. Monitor blood counts and do not give Lomustine Capsules more frequently than every 6 weeks. ( 2.2 , 2.3 , 5.1 ) Risk of Overdosage PRESCRIBE, DISPENSE, AND ADMINISTER ONLY ENOUGH CAPSULES FOR ONE DOSE. Fatal toxicity occurs with overdosage of Lomustine Capsules. Both physician and pharmacist should emphasize to patient that only one dose of Lomustine Capsules is taken every 6 weeks. ( 2.1 , 5.2 , 10 )
LOMUSTINE Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
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 LOMUSTINE's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications LOMUSTINE treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to LOMUSTINE
3 of 5FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
36 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT06325683 | NCI-2024-01995 NCI-2024-01995, A072201 | Ph 2 | suspended | Anti-Lag-3 (Relatlimab) and Anti-PD-1 Blockade (Nivolumab) Versus Standard of Care (Lomustine) for the Treatment of Patients With Recurrent Glioblastoma |
| NCT03970447 GBM AGILE | GCAR-7213 | Ph 2, Ph 3 | recruiting | A Trial to Evaluate Multiple Regimens in Newly Diagnosed and Recurrent Glioblastoma |
| NCT03382977 | VBI-1901-01 | Ph 1, Ph 2 | terminated | Study to Evaluate Safety, Tolerability, and Optimal Dose of Candidate GBM Vaccine VBI-1901 in Recurrent GBM Subjects |
| NCT05095376 | NRG-BN011 NCI-2021-10331, NRG-BN011 | Ph 3 | active not recruiting | Testing the Addition of the Chemotherapy Drug Lomustine (Gleostine) to the Usual Treatment (Temozolomide and Radiation Therapy) for Newly Diagnosed MGMT Methylated Glioblastoma |
| NCT02724579 | ACNS1422 NCI-2016-00150, ACNS1422 | Ph 2 | active not recruiting | Reduced Craniospinal Radiation Therapy and Chemotherapy in Treating Younger Patients With Newly Diagnosed WNT-Driven Medulloblastoma |
| NCT05382338 | ACNS2031 NCI-2022-04866, ACNS2031 | Ph 3 | recruiting | A Study of Treatment for Medulloblastoma Using Sodium Thiosulfate to Reduce Hearing Loss |
| NCT05902169 SONOBIRD | SC9-GBM-03 2023-505829-14-00 | Ph 3 | recruiting | Sonocloud-9 in Association With Carboplatin Versus Standard-of-Care Chemotherapies (CCNU or TMZ) in Recurrent GBM |
| NCT06336291 GLIOSTELLA | PH-L19TNFLOM-01/23 | Ph 2 | active not recruiting | A Study With L19TNF in Combination With Lomustine in Patients With Glioblastoma at Progression or Recurrence |
| NCT04762069 | CNS-201 | Ph 2 | active not recruiting | A Study of Berubicin in Adult Subjects With Recurrent Glioblastoma Multiforme |
| NCT06419946 | 2023-506998-35-00 | Ph 3 | recruiting | Lomustine in Addition to Standard of Care in Patients With MGMT Methylated Glioblastoma |
| NCT03678883 | 1801 | Ph 2 | active not recruiting | 9-ING-41 in Patients With Advanced Cancers |
| NCT05904119 LEGATO | EORTC-2227-BTG | Ph 3 | recruiting | Lomustine With and Without Reirradiation for First Progression of Glioblastoma: a Randomized Phase III Study |
| NCT04049669 | GCC1949 R01CA229646 | Ph 2 | active not recruiting | Pediatric Trial of Indoximod With Chemotherapy and Radiation for Relapsed Brain Tumors or Newly Diagnosed DIPG |
| NCT01582269 results posted | 13849 H9H-MC-JBAL, 2011-004418-40 | Ph 2 | completed | A Study in Recurrent Glioblastoma (GB) |
| NCT04402073 PersoMed-I | 1634 | Ph 2 | terminated | Personalized Risk-Adapted Therapy in Post-Pubertal Patients With Newly-Diagnosed Medulloblastoma |
| NCT07145112 | UCDCC321 P30CA093373, NCI-2025-06916 | Ph 1 | recruiting | Laser Interstitial Thermal Therapy (LITT) and Lomustine (CCNU) for Recurrent Glioblastoma |
| NCT04863950 | CTMS# 20-0148 | Ph 2 | recruiting | Investigator-Initiated Study of Imipramine Hydrochloride and Lomustine in Recurrent Glioblastoma |
| NCT02986178 results posted | Pro00077024 | Ph 2 | completed | Lerapolturev in Recurrent Malignant Glioma |
| NCT05304663 GLIOASTRA | PH-L19TNFLOM-05/21 | Ph 1 | withdrawn | Safety and Efficacy of Different Administration Sequences of L19TNF With Lomustine in Glioblastoma at First Progression |
| NCT03425292 | JWCI-17-0801 | Ph 1 | completed | A Longitudinal Assessment of Tumor Evolution in Patients With Brain Cancer |
| NCT04933942 RIGOLETTO | EORTC-1926-BTG | Ph 2 | withdrawn | Phase II Trial of Romiplostim for Thrombocytopenia Induced by Lomustine at First Progression of MGMT Promoter-meth Glioblastoma |
| NCT01775475 results posted | AMC-068 NCI-2012-01695, U01CA121947 | Ph 2 | completed | Intravenous Chemotherapy or Oral Chemotherapy in Treating Patients With Previously Untreated Stage III-IV HIV-Associated Non-Hodgkin Lymphoma |
| NCT02926222 REGOMA | IOV-GB-1-2014 REGOMA 2014-003722-41 | Ph 2 | completed | Regorafenib in Relapsed Glioblastoma |
| NCT03022578 results posted | 2016-0443 NCI-2018-01286, 2016-0443 | Ph 2 | terminated | Laser Interstitial Thermal Therapy and Lomustine in Treating Patients With Recurrent Glioblastoma or Anaplastic Astrocytoma |
| NCT02765165 results posted | P311-201 | Ph 1, Ph 2 | terminated | Phase 1/2 Study of USL311 +/- Lomustine in Advanced Solid Tumors or Relapsed/Recurrent Glioblastoma Multiforme (GBM) |
| NCT01290939 | EORTC-26101 EORTC-26101, EU-21103 | Ph 3 | completed | Bevacizumab and Lomustine for Recurrent GBM |
| NCT01934361 | CBKM120E2102 2013-003129-27 | Ph 1 | completed | Phase Ib/II Study of Buparlisib Plus Carboplatin or Lomustine in Patients With Recurrent Glioblastoma Multiforme |
| NCT02343406 INTELLANCE-2 results posted | M14-483 2014-004438-24, EORTC 1410-BTG | Ph 2 | completed | Adult Study: ABT-414 Alone or ABT-414 Plus Temozolomide vs. Lomustine or Temozolomide for Recurrent Glioblastoma Pediatric Study: Evaluation of ABT-414 in Children With High Grade Gliomas |
| NCT01067469 results posted | 2009-0597 NCI-2011-00559 | Ph 2 | completed | Standard Dose Bevacizumab Versus Low Dose Bevacizumab Plus Lomustine (CCNU) for Recurrent Glioblastoma Multiforme (GBM) |
| NCT02414165 Toca5 | Tg 511-15-01 FD-R-5732 | Ph 2, Ph 3 | terminated | The Toca 5 Trial: Toca 511 & Toca FC Versus Standard of Care in Patients With Recurrent High Grade Glioma |
| NCT01860638 | MO28347 2012-003138-17 | Ph 2 | completed | A Comparison of Continuous Bevacizumab (Avastin) Treatment or Placebo in Addition to Lomustine Followed by Standard of Care After Disease Progression in Participants With Glioblastoma |
| NCT01989052 | Pro00047969 | Ph 1 | terminated | Ph 1/2 CTO With Lomustine for Bevacizumab-Naive Recurrent Glioma |
| NCT01563614 | DepoRaCe | Ph 1 | terminated | A Clinical Trial to Assess the Safety & Efficacy of the Treatment of Patients With Metastasis From Malignant Melanoma - Treatment Consists of the Substances Lomustine (Capsules) & Cytarabine (Injected Into an Area Near the Spinal Cord), Accompanied by Radiotherapy of the Brain |
| NCT00761280 SAPPHIRE results posted | AP 12009-G005 | Ph 3 | terminated | Efficacy and Safety of AP 12009 in Patients With Recurrent or Refractory Anaplastic Astrocytoma or Secondary Glioblastoma |
| NCT00590837 LAM-SA 2007 | CHUBX 2007/13 | Ph 3 | completed | Adding Lomustine to Chemotherapy in Older Patients With Acute Myelogenous Leukemia (AML), and Allogeneic Transplantation for Patients From 60 to 65 Years Old |
| NCT00948389 results posted | CA180-274 Protocol 26083, 2009-010576-21 | Ph 1, Ph 2 | terminated | Study of CCNU (Lomustine) Plus Dasatinib in Recurrent Glioblastoma (GBM) |
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.
LOMUSTINE FDA Label Details
Indications & Usage
LOMUSTINE is indicated for the treatment of Brain Tumor; Hodgkin's Lymphoma.
WARNING: DELAYED MYELOSUPPRESSION and RISK OF OVERDOSAGE DELAYED MYELOSUPPRESSION Lomustine Capsules cause myelosuppression including fatal myelosuppression. Myelosuppression is delayed, dose-related, and cumulative; occurring 4 to 6 weeks after drug administration and persisting for 1 to 2 weeks. T...
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GLEOSTINE
Full clinical data, patents, trials, and competitive landscape for lomustine.
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