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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
Cytarabine Acute leukemia

Meningeal leukemia

Chronic myelogenous 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
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