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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

rhabdomyosarcoma

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
 

Philadelphia chromosome-positive acute lymphoblastic 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