Drugs Approved for Childhood Cancers

This page lists cancer drugs approved by the Food and Drug Administration (FDA) for use in children. The list includes generic and brand names. The drug names link to NCI’s Cancer Drug Information summaries. There are drugs and drug combinations used to treat children with cancer that are not listed here.

Drugs Approved for Alveolar Soft Part Sarcoma

Drugs Approved for Colorectal Cancer

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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

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Drugs Approved for Epithelioid Sarcoma

Tazemetostat Hydrobromide (Tazverik)

Drugs Approved for Ewing Sarcoma

Dactinomycin (Cosmegen)

Drugs Approved for Gestational Trophoblastic Disease

Dactinomycin (Cosmegen)

Drugs Approved for Giant Cell Astrocytoma

Drugs Approved for Glioma

Drugs Approved for Hodgkin Lymphoma

Drugs Approved for Leukemia

Drugs Approved for Acute Lymphoblastic Leukemia (ALL)

Drugs Approved for Acute Myeloid Leukemia (AML)

Drugs Approved for Blastic Plasmacytoid Dendritic Cell Neoplasm

Drugs Approved for Chronic Myelogenous Leukemia (CML)

Drugs Approved for Juvenile Myelomonocytic Leukemia (JMML)

Drugs Approved for Melanoma

Drugs Approved for Merkel Cell Carcinoma

Drugs Approved for Neuroblastoma

Drugs Approved for Neurofibromatosis Type 1

Drugs Approved for Non-Hodgkin Lymphoma

Drugs Approved for Pheochromocytoma and Paraganglioma

Drugs Approved for Rhabdomyosarcoma

Drugs Approved for Solid Tumors Anywhere in the Body

Drugs Approved for Thyroid Cancer

Drugs Approved for Wilms Tumor and Other Childhood Kidney Cancers

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