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 |
| Cytarabine | Acute leukemia Meningeal leukemia Chronic myelogenous 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 |
| Procarbazine Hydrochloride* | Hodgkin 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 |