FDA-Approved Drugs for Childhood Cancer
The Food and Drug Administration has approved more than two dozen drugs to treat childhood cancers. Data from NCI-sponsored clinical trials were used to support the approvals of several of these drugs (marked by asterisks).
| Drug Name | Childhood Cancer |
|---|---|
| Asparaginase Erwinia chrysanthemi* | acute lymphoblastic leukemia |
| Avelumab | Merkel cell carcinoma |
| Blinatumomab | recurrent or refractory B-cell acute lymphoblastic leukemia |
| Calaspargase Pegol-mknl* | acute lymphoblastic leukemia |
| Clofarabine* | acute lymphoblastic leukemia |
| Cyclophosphamide | acute lymphoblastic leukemia |
| Dactinomycin |
Ewing sarcoma Wilms tumor metastatic nonseminomatous testicular cancer |
| Dasatinib* | Philadelphia chromosome-positive chronic myelogenous leukemia |
| Daunorubicin Hydrochloride | acute lymphoblastic leukemia |
| Dinutuximab (monoclonal antibody ch14.18)* | neuroblastoma |
| Everolimus | subependymal giant cell astrocytoma |
| Gemtuzumab Ozogamicin | CD33-positive acute myeloid leukemia |
| Imatinib* |
chronic myelogenous leukemia |
| Iobenguane I-131 |
malignant pheochromocytoma paraganglioma |
| Ipilimumab |
melanoma mismatch repair-deficient and microsatellite instability-high colorectal cancer |
| Larotrectinib* | solid tumors with an NTRK gene fusion |
| Mercaptopurine | acute lymphoblastic leukemia |
| Nelarabine* |
T-cell acute lymphoblastic leukemia T-cell lymphoblastic lymphoma |
| Nilotinib | Philadelphia chromosome-positive chronic myelogenous leukemia |
| Nivolumab | mismatch repair-deficient and microsatellite instability-high colorectal cancer |
| Pegaspargase* | acute lymphoblastic leukemia |
| Pembrolizumab* |
refractory classic Hodgkin lymphoma mismatch repair-deficient and microsatellite instability-high solid tumors recurrent, locally advanced, or metastatic Merkel cell carcinoma primary mediastinal large B-cell lymphoma |
| Tagraxofusp-erzs | blastic plasmacytoid dendritic cell neoplasm |
| Teniposide (discontinued) | acute lymphoblastic leukemia |
| Tisagenlecleucel* | refractory B-cell acute lymphoblastic leukemia |
| Vincristine Sulfate |
acute leukemia Hodgkin lymphoma neuroblastoma non-Hodgkin lymphoma rhabdomyosarcoma Wilms tumor |