Phuturdex › Diseases › Neuroblastoma
Drugs in development · updated 30 October 2026
Neuroblastoma: drugs in development
As of 30 October 2026 there are 90 active clinical trials (recruiting or ongoing, phases I–III) on 95 drugs for neuroblastoma. 24 are experimental, that is not yet approved in the United States.
Summary updated every week · data as of 30 October 2026. In Phuturdex you get the full detail of every drug and an email when an alert is published on the ones you follow.
Sign in free →Experimental drugs
Not yet approved by the US FDA for any indication.
| Drug | Most advanced phase | Active trials | Reference trial |
|---|---|---|---|
| GM-CSFGranulocyte-Macrophage Colony-Stimulating Factor | I–II | 6 | NCT01757626 |
| Tipifarnib | II | 3 | NCT04284774 |
| OPT-821 | II | 2 | NCT06057948 |
| Ulixertinib | II | 2 | NCT03698994 |
| AMXT 1501 Dicaprate | I–II | 1 | NCT06465199 |
| APG-115 | I–II | 1 | NCT05701306 |
| ChidamideTucidinostat, HBI-8000 | II | 1 | NCT07318831 |
| LisaftoclaxAPG-2575, Lisafotoclax | I–II | 1 | NCT05701306 |
| MIBG | II | 1 | NCT02378428 |
| PEEL-224 | I–II | 1 | NCT06721689 |
| SamotolisibLY3023414 | II | 1 | NCT03155620 |
| Silmitasertib | I–II | 1 | NCT06541262 |
| SintilimabSinitilimab, Sindilimab | II | 1 | NCT06013618 |
| Tegavivint | I–II | 1 | NCT04851119 |
| Zilovertamab vedotin | I–II | 1 | NCT06395103 |
| ADCT-701 | I | 1 | NCT06041516 |
| Becotatug VedotinMRG003 | I | 1 | NCT07584499 |
| Iopofosine I 131CLR 131 | I | 1 | NCT03478462 |
| Irinotecan SucrosofateMM-398 | I | 1 | NCT02013336 |
| LY3295668 Erbumine | I | 1 | NCT04106219 |
| RhIL-15 | I | 1 | NCT06995872 |
| VAL-413 | I | 1 | NCT04337177 |
| 18F-MFBG | Early I | 1 | NCT05069220 |
| IR-101 | Early I | 1 | NCT07067346 |
Sorted by most advanced phase and number of active trials.
Approved drugs in new trials
Approved by the US FDA for at least one indication, not necessarily this disease, and now in active trials on it.
| Drug | Most advanced phase | Active trials | Reference trial |
|---|---|---|---|
| CyclophosphamideCyclophosphamid, Cytoxan | III | 22 | NCT06172296 |
| TemozolomideTemodal, Temodar | III | 17 | NCT06172296 |
| IrinotecanOnivyde, CPT-11 | III | 14 | NCT06172296 |
| DinutuximabDinituximab, Ch14.18 | III | 13 | NCT06172296 |
| TopotecanHycamtin®, Topotecan Hospira® | III | 10 | NCT06172296 |
| EtoposideAvopef®, Etopophos Preservative Free® | III | 8 | NCT06172296 |
| CarboplatinCarboplatine, Paraplatin | III | 7 | NCT06172296 |
| VincristineVincristin, Oncovin [vincristine] | III | 7 | NCT06172296 |
| SargramostimrhuGM-CSF, leukine | III | 6 | NCT03126916 |
| DoxorubicinAdriamycin, Doxorubincin | III | 5 | NCT06172296 |
| IsotretinoinAbsorica Ld®, Absorica® | III | 5 | NCT06172296 |
| MelphalanEvomela®, Hepzato® | III | 5 | NCT06172296 |
| ThiotepaTepylute®, Tepadina And Sodium Chloride® | III | 5 | NCT06172296 |
| CisplatinCis Platinum, Cisplatine | III | 3 | NCT06172296 |
| BusulfanBusulphan, Busulfex® | III | 2 | NCT03126916 |
Sorted by most advanced phase and number of active trials. Showing 15 of 71 drugs.
All 95 drugs, with the details
In Phuturdex every drug has all its trials, sponsors and countries, FDA and EMA approvals, orphan designations and safety alerts. Free until 31 March 2027.
Open in Phuturdex →Specific forms
- Esthesioneuroblastoma 10 drugs
- Sinonasal undifferentiated carcinoma 7 drugs
- Melanoma 352 drugs
- Medullary carcinoma 13 drugs
- Bladder neoplasm 185 drugs
- Testicular Neoplasms 17 drugs
- Glioblastoma 245 drugs
- Uterine cervix neoplasm 224 drugs
- Non-small-cell lung carcinoma 726 drugs
- Merkel cell carcinoma 48 drugs
- Chondrosarcoma 13 drugs
- Nasopharyngeal Carcinoma 71 drugs
Broader categories
- Glandular and epithelial neoplasm 12 drugs
- Neuroectodermal tumor 3 drugs
- Nerve tissue neoplasm 4 drugs
- Neuroepithelial neoplasm 4 drugs
- Germ cell and embryonal neoplasms 35 drugs
- Primitive neuroectodermal tumor 31 drugs
Source: Phuturdex analysis of ClinicalTrials.gov data (U.S. National Library of Medicine), updated 30 October 2026. Active trials = recruiting or ongoing, phases I–III, with at least one identified drug; phase and sites refer to the trials on this disease. Disease name: Wikidata; NLM MeSH term: D009447 “Neuroblastoma”. Prevalence: Orphanet.