THALIDOMIDE
Thalidomide helps patients with newly diagnosed multiple myeloma when used in combination with the medication dexamethasone. It also treats the skin-related symptoms of moderate to severe erythema nodosum leprosum and is used as a long-term therapy to prevent these symptoms from returning. While it manages these specific skin manifestations, it is not used as a standalone treatment for patients who also have moderate to severe nerve inflammation.
How THALIDOMIDE Works
This drug works by targeting cereblon, a protein that is part of an enzyme complex involved in regulating cellular activities. By binding to this target, it modifies the immune system and reduces inflammation, specifically by suppressing excessive tumor necrosis factor-alpha and increasing the activity of natural killer cells. It also helps slow disease progression by inhibiting the growth of new blood vessels and stopping the proliferation of endothelial cells.
Details
- Status
- Prescription
- First Approved
- 2023-04-27
- Routes
- ORAL
- Dosage Forms
- CAPSULE
THALIDOMIDE Approval History
What THALIDOMIDE Treats
2 indicationsTHALIDOMIDE is approved for 2 conditions since its original approval in 2023. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Multiple Myeloma
- Erythema Nodosum Leprosum
THALIDOMIDE Boxed Warning
EMBRYO-FETAL TOXICITY AND VENOUS THROMBOEMBOLISM WARNING: EMBRYO-FETAL TOXICITY AND VENOUS THROMBOEMBOLISM See full prescribing information for complete boxed warning. EMBRYO -FETAL TOXICITY • If THALOMID is taken during pregnancy, it can cause severe birth defects or embryo-fetal death. THALOMID should never be used by females who are pregnant or who could be pregnant while taking the drug. Even a single dose [1 capsule (regardless of strength)] taken by a pregnant woman during her pregnancy ca...
WARNING: EMBRYO-FETAL TOXICITY AND VENOUS THROMBOEMBOLISM WARNING: EMBRYO-FETAL TOXICITY AND VENOUS THROMBOEMBOLISM See full prescribing information for complete boxed warning. EMBRYO -FETAL TOXICITY • If THALOMID is taken during pregnancy, it can cause severe birth defects or embryo-fetal death. THALOMID should never be used by females who are pregnant or who could be pregnant while taking the drug. Even a single dose [1 capsule (regardless of strength)] taken by a pregnant woman during her pregnancy can cause severe birth defects. • Pregnancy must be excluded before start of treatment. Prevent pregnancy thereafter by the use of two reliable methods of contraception. ( 5.1 , 8.3 ) THALOMID is only available through a restricted distribution program, the THALOMID REMS ® program ( 5.2 ). VENOUS THROMBOEMBOLISM • Significant increased risk of deep vein thrombosis (DVT) and pulmonary embolism (PE) in patients with multiple myeloma receiving THALOMID with dexamethasone ( 5.3 ). EMBRYO-FETAL TOXICITY If THALOMID is taken during pregnancy, it can cause severe birth defects or embryo-fetal death. THALOMID should never be used by females who are pregnant or who could become pregnant while taking the drug. Even a single dose [1 capsule (regardless of strength)] taken by a pregnant woman during her pregnancy can cause severe birth defects. Because of this toxicity and in an effort to make the chance of embryo-fetal exposure to THALOMID as negligible as possible, THALOMID is approved for marketing only through a special restricted distribution program: THALOMID REMS program, approved by the Food and Drug Administration. Information about THALOMID and the THALOMID REMS program is available at www.thalomidrems.com or by calling the REMS Call Center at 1-888-423-5436. VENOUS THROMBOEMBOLISM The use of THALOMID in multiple myeloma results in an increased risk of venous thromboembolism, such as deep venous thrombosis and pulmonary embolism. This risk increases significantly when TH
THALIDOMIDE 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.
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 THALIDOMIDE's indications
See all emerging drugs →Phase 3 candidates targeting molecules with no FDA-approved drug, in indications THALIDOMIDE treats. First-in-class if their pivotal trials read out positive.
Drugs Similar to THALIDOMIDE
3 of 20FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
47 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT00871013 | 108053 | Ph 2 | active not recruiting | Trial for Patients Not Qualifying for TT4 and TT5 Protocols Because of Prior Therapy |
| NCT00602641 results posted | NCI-2009-00522 NCI-2009-00522, ECOG-E1A06 | Ph 3 | active not recruiting | Melphalan, Prednisone, and Thalidomide or Lenalidomide in Treating Patients With Newly Diagnosed Multiple Myeloma |
| NCT03069326 | 16-1498 | Ph 2 | active not recruiting | A Clinical Study to Test the Effects of Ruxolitinib And Thalidomide Combination for Patients With Myelofibrosis |
| NCT06617182 | KY20242229-F-1 | Ph 2 | recruiting | Efficacy and Safety of Thalidomide Combined With Glutamine in the Treatment of Radiation Intestinal Injury. |
| NCT01356290 MEMMAT | MUV-MEMMAT-01 | Ph 2 | recruiting | Antiangiogenic Therapy for Children With Recurrent Medulloblastoma, Ependymoma, ATRT and Rare CNS Tumors |
| NCT02858804 BDH-MCL01 | IIT2015007 | Ph 4 | completed | EDOCH Alternating With DHAP for New Diagnosed Younger MCL |
| NCT02844309 BDH-WM02 | IIT2015004 | Ph 4 | completed | The Efficacy of TCD Following by TP Maintenance Therapy in Newly Diagnosed WM |
| NCT07172659 | MDGH-DOV-2001 | Ph 2 | not yet recruiting | Efficacy and Safety Study of Dovramilast in People With Leprosy Type 2 Reaction |
| NCT03004287 | 206241 | Ph 2 | active not recruiting | 2015-12: A Study Exploring the Use of Early and Late Consolidation/Maintenance Therapy |
| NCT01659658 results posted | C16011 2011-005468-10, U1111-1164-7621 | Ph 3 | terminated | Study of Dexamethasone Plus IXAZOMIB (MLN9708) or Physicians Choice of Treatment in Relapsed or Refractory Systemic Light Chain (AL) Amyloidosis |
| NCT00869232 | 106952 | Ph 2 | active not recruiting | UARK 2008-02 A Trial for High-risk Myeloma Evaluating Accelerating and Sustaining Complete Remission |
| NCT06490627 BTM | NIBD/IRB-266/01-2024 | Ph 2 | recruiting | Unraveling the Impact of Thalidomide at Diverse Doses in Transfusion Dependent Beta Thalassemia |
| NCT06490601 Thalidomide | NIBD/IRB-242/12-2022 | Ph 2 | active not recruiting | Long Term Beta Thalassemia Treatment: Findings From The Extension Period |
| NCT01998971 | CR103015 54767414MMY1001, 2013-003491-12 | Ph 1 | completed | A Study of JNJ-54767414 (HuMax CD38) (Anti-CD38 Monoclonal Antibody) in Combination With Backbone Treatments for the Treatment of Patients With Multiple Myeloma |
| NCT01539083 VCAT results posted | CR018751 26866138-MMY-2073 | Ph 3 | completed | Velcade (Bortezomib) Consolidation After Transplant |
| NCT02507336 results posted | 20150402 | Ph 2 | completed | Long-Term Followup and/or Thalidomide Maintenance Therapy for Patients Enrolled on Clinical Trial 20030165 |
| NCT04352205 | STUDY00000007 NCI-2020-01133, WINSHIP4949-20 | Ph 2 | terminated | Daratumumab-Based Therapy for the Treatment of Newly Diagnosed Multiple Myeloma With Kidney Failure |
| NCT02586038 | UNITO-EMN10 | Ph 2 | completed | STUDY THAT COMPARE 3 ARM: MLN9708 DEXAMETHASONE, MLN9708 CYCLOPHOSPHAMIDE AND DEXAMETHASONE, MLN9708 THALIDOMIDE AND DEXAMETHASONE FOLLOWED BY MAINTENANCE WITH MLN9708 IN NEWLY DIAGNOSED ELDERLY MULTIPLE MYELOMA PATIENTS |
| NCT02891811 | AGMT_MM-2 2016-000475-24 | Ph 2 | completed | Patients With Newly Diagnosed Multiple Myeloma Comparing KTd vs. KRd Induction Therapy and Investigating a K-mono Maintenance Strategy |
| NCT06268093 | XWTETS | Ph 2 | recruiting | The Therapeutic Effect of Thalidomide in Syringomyelia |
| NCT01661400 ASCR | 201209088 | Ph 1 | completed | Anti-Angiogenic Therapy Post Transplant (ASCR) for Pediatric Solid Tumors |
| NCT06146478 | Dir/KMU-EB/TC/000863 | Ph 3 | completed | Deciphering Effects of Thalidomide on Red Blood Cells in Transfusion Dependents Beta Thalassemia Patients |
| NCT06017284 PTCA199-5 | PTCA199-5 | Ph 3 | recruiting | Thalidomide to Chemotherapy Related Nausea and Vomiting in Pancreatic Cancer |
| NCT01849783 results posted | 201208775 10120146, P30CA086862 | Ph 2 | completed | Autologous Stem Cell Transplant Followed By Maintenance Therapy in Treating Elderly Patients With Multiple Myeloma |
| NCT02424851 OPTIMAL results posted | 26866138-MMY2070 2012-003947-31 | Ph 2 | completed | Optimising Renal Outcome in Myeloma Renal Failure |
| NCT01125176 ThRiL results posted | 1112012062 RV-CLL-PI-0391 | Ph 2 | completed | Alternating Thalidomide and Lenalidomide Therapy Plus Rituximab (ThRiL) as Initial Treatment for Patients With CLL |
| NCT03122431 results posted | HPLC-Rheumatic diseases | Ph 4 | completed | Relevance of Monitoring Blood and Salivar Levels of Drugs Used in Rheumatic Autoimmune Diseases |
| NCT00792142 results posted | 06143 P30CA033572, CHNMC-06143 | Ph 2 | completed | Bortezomib, Thalidomide, and Dexamethasone After Melphalan and Stem Cell Transplant in Treating Patients With Stage I-III Multiple Myeloma |
| NCT02545907 CATALYST results posted | 14/0786 | Ph 1, Ph 2 | completed | A Dose Escalation Study of Carfilzomib Taken With Thalidomide and Dexamethasone in Relapsed AL Amyloidosis |
| NCT00966693 results posted | 2009-0179 NCI-2018-01857, 2009-0179 | Ph 1, Ph 2 | completed | Lenalidomide, Thalidomide and Dexamethasone in Treating Participants With Relapsed or Refractory Multiple Myeloma |
| NCT03221166 | NET-2013-02355002 | Ph 3 | terminated | Thalidomide Versus Infliximab in New Onset Crohn's Disease With Poor Prognostic Factors |
| NCT03608501 IDEALL | C16041 U1111-1214-1078 | Ph 2 | withdrawn | A Study of Ixazomib, Thalidomide and Dexamethasone in Newly Diagnosed and Treatment-naive Multiple Myeloma (MM) Participants Non-eligible for Autologous Stem-cell Transplantation |
| NCT02410694 | AGMT_MM-1 | Ph 2 | completed | Ixazomib in Combination With Thalidomide - Dexamethasone in Patients With Relapsed and/or Refractory Multiple Myeloma |
| NCT01779167 THRiL for WM results posted | 1112012086 RV-WM-PI-0690 | Ph 2 | terminated | Thalidomide, Lenalidomide, and Rituximab for Previously Treated Waldenstrom Macroglobulinemia |
| NCT01526694 | BDT-01-2011 | Ph 2 | completed | Phase II Trial Designed to Determine Efficacy and Safety of Bendamustine+Dexamethasone+Thalidomide in R/R MM |
| NCT01057225 results posted | MC0982 NCI-2009-01699, PT-171-502 | Ph 1, Ph 2 | completed | Cyclophosphamide, Carfilzomib, Thalidomide, and Dexamethasone in Treating Patients With Newly Diagnosed Active Multiple Myeloma |
| NCT01485224 THALI-HHT | EudratCT 2011-004096-36 | Ph 2 | completed | Efficacy of Thalidomide in the Treatment of Hereditary Hemorrhagic Telangiectasia |
| NCT01548573 results posted | 201202818 7R01CA115399 | Ph 2 | terminated | Tandem Auto Transplantation in Myeloma Patients With <12 Months of Prior Treatment |
| NCT01632150 results posted | CA204-010 2011-005121-49 | Ph 2 | completed | Safety Study of Elotuzumab in Combination With Thalidomide and Dexamethasone in Relapsed and/or Refractory Multiple Myeloma |
| NCT02203253 | CLOG1302 | Ph 3 | completed | Efficacy of Thalidomide in Preventing Chemotherapy-induced Delayed Nausea and Vomiting |
| NCT02754960 | rjyyxhk57809 | Ph 2 | withdrawn | Efficacy Study of Thalidomide in Gastrointestinal Vascular Malformation Related Bleeding |
| NCT02301949 | rjyyxhnj3015 | Ph 2 | withdrawn | Retreatment and Its Efficiency of Thalidomide for Vascular Malformation Patients With Failure of First Course Treatment |
| NCT01976195 | ITP-Thalidomide | Ph 2 | withdrawn | High-dose Dexamethasone Combining Thalidomide Versus Dexamethasone Mono-therapy for Management of Newly-diagnosed ITP |
| NCT02501291 | IBD201501 | Ph 2 | completed | Thalidomide in Treating Crohn's Disease |
| NCT01055301 | S0833 S0833, U10CA032102 | Ph 2 | withdrawn | S0833, Bortezomib, Thalidomide, Lenalidomide, Combination Chemotherapy, and Autologous Stem Cell Transplant in Treating Patients With Newly Diagnosed Multiple Myeloma |
| NCT00723658 | S0629 S0629, U10CA032102 | Ph 2 | withdrawn | S0629, Observation or Combination Chemotherapy, Bortezomib, Thalidomide, and Rituximab Followed By Two Autologous Peripheral Blood Stem Cell Transplants in Treating Patients With Waldenstrom Macroglobulinemia |
| NCT00720538 TALIBDP | TAL-005414-20 | Ph 3 | completed | Thalidomide in Pediatric Inflammatory Bowel Diseases. |
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.
THALIDOMIDE FDA Label Details
Indications & Usage
THALIDOMIDE is indicated for the treatment of Multiple Myeloma; Erythema Nodosum Leprosum.
WARNING: EMBRYO-FETAL TOXICITY AND VENOUS THROMBOEMBOLISM WARNING: EMBRYO-FETAL TOXICITY AND VENOUS THROMBOEMBOLISM See full prescribing information for complete boxed warning. EMBRYO -FETAL TOXICITY • If THALOMID is taken during pregnancy, it can cause severe birth defects or embryo-fetal death. TH...
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THALOMID
Full clinical data, patents, trials, and competitive landscape for thalidomide.
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.