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Pediatric Oncology Drug Approvals

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Since the early 1950s, the FDA has approved more than 60 drugs with over 85 associated indications for the treatment of pediatric cancers. The number of oncology products approved for use in pediatric patients with cancer has been expanding due to emerging scientific discoveries and changes in the regulatory framework to accelerate pediatric drug development.

These approvals reflect a variety of strategies applied to pediatric drug development. Examples include initial approvals of drugs developed specifically for treatment of pediatric cancers, supplemental approvals of drugs initially developed for adult cancers, approvals based on programs employing extrapolation of adult efficacy data to pediatric populations, and approvals of tumor-agnostic indications.

Diverse therapeutic agents such as small-molecule kinase inhibitors, monoclonal antibodies and other antibody-based therapies (e.g., bispecific T-cell engager, antibody-drug conjugate), and genetically engineered cell therapy (CAR T-cell) are now part of the growing pediatric oncology armamentarium along with cytotoxic chemotherapy drugs.

Each row on the table represents an FDA-approved pediatric indication and includes the drug generic and U.S. brand names (allowing for the fact that some brand names are discontinued over time), the indication as listed in the most recent product labeling, and the original FDA pediatric approval date for that indication. Drug names link to Drugs@FDA or CBER’s Novel Biological Products to provide additional information.

The accompanying downloadable file includes additional information for each drug/indication pair including the drug target and pharmacologic class, the clinical trials that supported approval, the pediatric age groups, and whether pediatric approvals were supplemental or corresponded to the initial drug approval in the U.S. When accessible, pediatric approval dates link to FDA approval notifications and to other resources for information on FDA-approved drugs.

The table below and the downloadable file are current as of November 2024. Updates will be made as new information becomes available.

Downloadable file for: Pediatric Approvals Additional Information

DRUGS APPROVED FOR PEDIATRIC CANCERS [BRAND NAME] 1 INDICATION 2 PEDIATRIC APPROVAL DATE 3*
Revumenib [Revuforj] Treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients 1 year and older 11-15-2024
Vorasidenib [Voranigo] Treatment of adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 (IDH1) or isocitrate dehydrogenase-2 (IDH2) mutation following surgery including biopsy, sub-total resection, or gross total resection 08-06-2024
Inotuzumab ozogamicin [Besponsa] Treatment of relapsed or refractory CD22-positive B-cell precursor acute lymphoblastic leukemia (ALL) in adult and pediatric patients 1 year and older 03-06-2024
Lutetium Lu 177 dotatate [Lutathera] Treatment of adult and pediatric patients 12 years and older with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut neuroendocrine tumors 04-23-2024
Tovorafenib [Ojemda] Treatment of patients 6 months of age and older with relapsed or refractory pediatric low-grade glioma (LGG) harboring a BRAF fusion or rearrangement, or BRAF V600 mutation 04-23-2024
Selpercatinib [Retevmo] Treatment of adult and pediatric patients 2 years of age and older with locally advanced or metastatic solid tumors with a RET gene fusion, as detected by an FDA-approved test, that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options 05-29-2024
Repotrectinib [Augtyro] Treatment of adult and pediatric patients 12 years of age and older with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion, are locally advanced or metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory alternative therapy 06-13-2024
Blinatumomab [Blincyto] Treatment of CD19-positive Philadelphia chromosome-negative B-cell precursor acute lymphoblastic leukemia (ALL) in the consolidation phase of multiphase chemotherapy in adult and pediatric patients one month and older 06-14-2024
Eflornithine [Iwilfin] To reduce the risk of relapse in adult and pediatric patients with high-risk neuroblastoma (HRNB) who have demonstrated at least a partial response to prior multiagent, multimodality therapy including anti-GD2 immunotherapy 12-13-2023
Bosutinib [Bosulif] Treatment of adult and pediatric patients 1 year of age and older with chronic phase (CP) Philadelphia chromosome-positive chronic myelogenous leukemia (Ph+ CML), newly-diagnosed or resistant or intolerant to prior therapy 09-26-2023
Dabrafenib [Tafinlar] In combination with trametinib, for the treatment of adult and pediatric patients 1 year of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options 8-31-2023 *
Dabrafenib [Tafinlar] In combination with trametinib, for the treatment of pediatric patients 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy 03-16-2023
Trametinib [Mekinist] In combination with dabrafenib, for the treatment of adult and pediatric patients 1 year of age and older with unresectable or metastatic solid tumors with BRAF V600E mutation who have progressed following prior treatment and have no satisfactory alternative treatment options 8-31-2023 *
Trametinib [Mekinist] In combination with dabrafenib, for the treatment of pediatric patients 1 year of age and older with low-grade glioma (LGG) with a BRAF V600E mutation who require systemic therapy 03-16-2023
Nivolumab [Opdivo] As a single agent or in combination with ipilimumab, for the treatment of adult and pediatric patients 12 years and older with unresectable or metastatic melanoma 02-15-2023
Nivolumab [Opdivo] As a single agent or in combination with ipilimumab, for the treatment of adult and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan 7-10-2018 *
Atezolizumab [Tecentriq] As a single agent, for the treatment of adult and pediatric patients 2 years of age and older with unresectable or metastatic alveolar soft part sarcoma (ASPS) 12-09-2022
Brentuximab vedotin [Adcetris] Treatment of pediatric patients 2 years and older with previously untreated high risk classical Hodgkin lymphoma (cHL), in combination with doxorubicin, vincristine, etoposide, prednisone, and cyclophosphamide 11-10-2022
Azacitidine [Vidaza] Treatment of pediatric patients aged 1 month and older with newly diagnosed Juvenile Myelomonocytic Leukemia (JMML) 05-20-2022
Nivolumab and Relatlimab-rmbw) [Opdualag] Treatment of adult and pediatric patients 12 years of age or older with unresectable or metastatic melanoma 03-18-2022
Pembrolizumab [Keytruda] Adjuvant treatment of adult and pediatric (12 years and older) patients with Stage IIB, IIC, or III melanoma following complete resection 12-03-2021
Rituximab [Rituxan] Treatment of pediatric patients aged 6 months and older with previously untreated, advanced stage, CD20-positive diffuse large B-cell lymphoma (DLBCL), Burkitt lymphoma (BL), Burkitt-like lymphoma (BLL) or mature B-cell acute leukemia (B-AL) in combination with chemotherapy 12-02-2021
Cabozantinib [Cabometyx] Treatment of adult and pediatric patients 12 years of age and older with locally advanced or metastatic differentiated thyroid cancer (DTC) that has progressed following prior VEGFR-targeted therapy and who are radioactive iodine-refractory or ineligible 09-17-2021
Asparaginase Erwinia chrysanthemi (recombinant)-rywn [Rylaze] As a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients 1 month or older who have developed hypersensitivity to E. coli-derived asparaginase 06-30-2021
Daunorubicin and cytarabine liposome [Vyxeos] Treatment of newly-diagnosed therapy-related acute myeloid leukemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC) in adults and pediatric patients 1 year and older 03-30-2021
Crizotinib [Xalkori] Treatment of pediatric patients 1 year of age and older and young adults with relapsed or refractory, systemic anaplastic large cell lymphoma (ALCL) that is ALK-positive 01-14-2021
Crizotinib [Xalkori] Treatment of adult and pediatric patients 1 year of age and older with unresectable, recurrent, or refractory inflammatory myofibroblastic tumor (IMT) that is ALK-positive 07-14-2022
Pralsetinib [Gavreto] Treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy 12-01-2020
Pralsetinib [Gavreto] Treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate) 12-01-2020
Naxitamab-gqgk [Danyelza] In combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy 11-25-2020
Pembrolizumab [Keytruda] Treatment of pediatric patients with refractory classical Hodgkin lymphoma (cHL), or cHL that has relapsed after 2 or more lines of therapy 10-14-2020 *
Gemtuzumab ozogamicin [Mylotarg] Treatment of newly-diagnosed CD33-positive acute myeloid leukemia in adults and pediatric patients 1 month and older 06-16-2020
Pembrolizumab [Keytruda] Treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase (mut/Mb)] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options 06-16-2020
Selpercatinib [Retevmo] Treatment of adult and pediatric patients 2 years of age and older with advanced or metastatic thyroid cancer with a RET gene fusion, as detected by an FDA-approved test, who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate) 5-29-2024 *
Selpercatinib [Retevmo] Treatment of adult and pediatric patients 2 years of age and older with advanced or metastatic medullary thyroid cancer (MTC) with a RET mutation, as detected by an FDA-approved test, who require systemic therapy 5-29-2024 *
Selumetinib [Koselugo] Treatment of pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN) 04-10-2020
Tazemetostat [Tazverik] Treatment of adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection 01-23-2020
Entrectinib [Rozlytrek] Treatment of adult and pediatric patients older than 1 month with solid tumors that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have progressed following treatment or have no satisfactory standard therapy 10-20-2023 *
Dasatinib [Sprycel] Treatment of pediatric patients 1 year of age and older with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) in combination with chemotherapy 12-21-2018
Tagraxofusp-erzs [Elzonris] Treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients 2 years and older 12-21-2018
Calaspargase pegol - mknl [Asparlas] As a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia in pediatric and young adult patients age 1 month to 21 years 12-20-2018
Pembrolizumab [Keytruda] Treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC) 12-19-2018
Larotrectinib [Vitrakvi] Treatment of adult and pediatric patients with solid tumors that have a neurotrophic receptor tyrosine kinase (NTRK) gene fusion without a known acquired resistance mutation, are metastatic or where surgical resection is likely to result in severe morbidity, and have no satisfactory alternative treatments or that have progressed following treatment 11-26-2018
Emapalumab-lzsg [Gamifant] Treatment of adult and pediatric (newborn and older) patients with primary hemophagocytic lymphohistiocytosis (HLH) with refractory, recurrent or progressive disease or intolerance with conventional HLH therapy 11-20-2018
Iobenguane I-131 [Azedra] Treatment of adult and pediatric patients 12 years and older with iobenguane scan positive, unresectable, locally advanced or metastatic pheochromocytoma or paraganglioma who require systemic anticancer therapy 07-30-2018
Ipilimumab [Yervoy] In combination with nivolumab, for the treatment of adult and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (mCRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan 07-10-2018
Pembrolizumab [Keytruda] Treatment of adult and pediatric patients with refractory primary mediastinal large B-Cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy 06-13-2018
Blinatumomab [Blincyto] Treatment of CD19-positive B-cell precursor acute lymphoblastic leukemia (ALL) in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1% in adult and pediatric patients one month and older 03-29-2018
Nilotinib [Tasigna] Treatment of adult and pediatric patients greater than or equal to 1 year of age with newly diagnosed Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase 03-22-2018
Nilotinib [Tasigna] Treatment of pediatric patients greater than or equal to 1 year of age with chronic phase and accelerated phase Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) with resistance or intolerance to prior tyrosine-kinase inhibitor (TKI) therapy 03-22-2018
Dasatinib [Sprycel] Treatment of pediatric patients 1 year of age and older with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in chronic phase 11-09-2017
Gemtuzumab ozogamicin [Mylotarg] Treatment of relapsed or refractory CD33-positive acute myeloid leukemia in adults and pediatric patients 2 years and older. 09-01-2017
Tisagenlecleucel [Kymriah] Treatment of patients up to 25 years of age with B-cell precursor acute lymphoblastic leukemia (ALL) that is refractory or in second or later relapse 08-30-2017
Nivolumab [Opdivo] Adjuvant treatment of adult and pediatric patients 12 years and older with completely resected Stage IIB, Stage IIC, Stage III, or Stage IV melanoma 10-13-2023 *
Ipilimumab [Yervoy] As a single agent or in combination with nivolumab, for the treatment of unresectable or metastatic melanoma in adult and pediatric patients 12 years and older 02-15-2023 *
Blinatumomab [Blincyto] Treatment of relapsed or refractory CD19-positive B-cell precursor acute lymphoblastic leukemia (ALL) in adult and pediatric patients one month and older 07-11-2017
Pembrolizumab [Keytruda] Treatment of adult and pediatric patients with unresectable or metastatic, microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options 05-23-2017
Avelumab [Bavencio] Treatment of adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (MCC) 03-23-2017
Dinutuximab [Unituxin] In combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), interleukin-2 (IL-2), and 13-cis-retinoic acid (RA), for the treatment of pediatric patients with high-risk neuroblastoma who achieve at least a partial response to prior first-line multiagent, multimodality therapy 03-10-2015
Denosumab [Xgeva] Treatment of adults and skeletally mature adolescents with giant cell tumor of bone that is unresectable or where surgical resection is likely to result in severe morbidity 06-13-2013
Imatinib Mesylate [Gleevec] & Pediatric patients with newly diagnosed Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL) in combination with chemotherapy 01-25-2013
Asparaginase Erwinia Chrysanthemi [Erwinaze] [currently in shortage] A component of a multi-agent chemotherapeutic regimen for the treatment of patients with acute lymphoblastic leukemia (ALL) who have developed hypersensitivity to E. coli-derived asparaginase 11-18-2011
Everolimus [Afinitor] & Adult and pediatric patients aged 1 year and older with tuberous sclerosis complex (TSC) for the treatment of subependymal giant cell astrocytoma (SEGA) that requires therapeutic intervention but cannot be curatively resected 10-29-2010
Imatinib Mesylate [Gleevec] & Newly diagnosed adult and pediatric patients with Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase 09-27-2006
Pegaspargase [Oncaspar] As a component of a multi-agent chemotherapeutic regimen for the first-line treatment of pediatric and adult patients with acute lymphoblastic leukemia (ALL) 07-24-2006
Nelarabine [Arranon] Treatment of T-cell acute lymphoblastic leukemia (T-ALL) and T-cell lymphoblastic lymphoma (T-LBL) in adult and pediatric patients age 1 year and older whose disease has not responded to or has relapsed following treatment with at least 2 chemotherapy regimens 10-28-2005
Clofarabine [Clolar] Treatment of pediatric patients 1 to 21 years old with relapsed or refractory acute lymphoblastic leukemia after at least two prior regimens 12-28-2004
Busulfan [Busulfex] For use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia 01-13-2003
Arsenic trioxide [Trisenox] Induction of remission and consolidation in patients with acute promyelocytic leukemia (APL) who are refractory to, or have relapsed from, retinoid and anthracycline chemotherapy, and whose APL is characterized by the presence of the t(15;17) translocation or PML/RAR-alpha gene expression 09-25-2000
Tretinoin [Vesanoid] Induction of remission in adults and pediatric patients 1 year of age and older with acute promyelocytic leukemia (APL) characterized by the presence of the t(15;17) translocation or PML/RARα gene expression, and who are refractory to or who have relapsed from anthracycline chemotherapy or for whom anthracycline-based chemotherapy is contraindicated 11-22-1995 #
Pegaspargase [Oncaspar] As a component of a multi-agent chemotherapeutic regimen for the treatment of pediatric and adult patients with ALL and hypersensitivity to native forms of L-asparaginase 02-01-1994
Daunorubicin Hydrochloride In combination with other approved anticancer drugs, for remission induction in acute lymphocytic leukemia of children and adults 12-19-1979
Asparaginase [Elspar] As a component of a multi-agent chemotherapeutic regimen for the treatment of patients with acute lymphoblastic leukemia (ALL) 01-10-1978
Lomustine [Gleostine] Treatment of patients with primary and metastatic brain tumors following appropriate surgical and/or radiotherapeutic procedures 08-04-1976
Lomustine [Gleostine] As a component of combination chemotherapy for the treatment of patients with Hodgkin's lymphoma whose disease has progressed following initial chemotherapy 08-04-1976
Doxorubicin Hydrochloride MULTIPLE 08-07-1974
Procarbazine Hydrochloride [Matulane] In combination with other anticancer drugs for the treatment of Stage III and IV Hodgkin's disease 07-22-1969
Cytarabine In combination with other approved anti-cancer drugs, for remission induction in acute non-lymphocytic leukemia of adults and pediatric patients 06-17-1969
Thioguanine [Tabloid] Remission induction and remission consolidation treatment of acute nonlymphocytic leukemias  
Vinblastine sulfate Palliative treatment of malignancies including generalized Hodgkin’s disease, histiocytosis X and testicular germ cell carcinoma 11-25-1965
Dactinomycin [Cosmegen] Treatment of adult and pediatric patients with Wilms tumor, as part of a multi-phase, combination chemotherapy regimen 12-10-1964
Dactinomycin [Cosmegen] Treatment of adult and pediatric patients with rhabdomyosarcoma, as part of a multi-phase, combination chemotherapy regimen 12-10-1964
Dactinomycin [Cosmegen] Treatment of adult and pediatric patients with Ewing sarcoma, as part of a multi-phase, combination chemotherapy regimen 12-10-1964
Dactinomycin [Cosmegen] Treatment of adult and pediatric patients with metastatic, nonseminomatous testicular cancer, as part of a multi-phase, combination chemotherapy regimen 12-10-1964
Vincristine Sulfate Acute leukemia; MULTIPLE (in combination)  
Cyclophosphamide [Cytoxan] MULTIPLE 11-16-1959
Methotrexate Treatment of adult and pediatric patients with acute lymphoblastic leukemia (ALL) as part of a combination chemotherapy regimen 12-07-1953
Methotrexate Prophylaxis and treatment of meningeal leukemia in adult and pediatric patients 12-07-1953
Methotrexate Treatment of adults and pediatric patients with Non-Hodgkin lymphoma 12-07-1953
Methotrexate Treatment of adults and pediatric patients with osteosarcoma as part of a combination chemotherapy regimen 12-07-1953
Mercaptopurine [Purinethol tablet] & Treatment of adult and pediatric patients with acute lymphoblastic leukemia (ALL) as part of a combination chemotherapy maintenance regimen 09-11-1953
  1. Column A [DRUGS APPROVED FOR PEDIATRIC CANCERS [BRAND NAME]: Reference products may have been discontinued. Links to generic products were selected based on extent of information and do not represent any specific product preference.
  2. Column B: [INDICATION]: This table reflects current data as of November 2024. To view additional information, please check the Drugs@FDA listing.
  3. Column C: [PEDIATRIC APPROVAL DATE]: Missing dates: Not available in digital records.

& Everolimus (Afinitor disperz: 08-29-2012); Mercaptopurine (Purixan suspension: 04-28-2014); Imatinib (Imkeldi oral solution: 11-22-2024)

⁕ The listed pediatric approval date reflects the most recent approval for the pediatric cancer(s) listed in the indication. Approved drugs for pediatric indications that were broadened or modified over time to extend the pediatric age group or approve the drug for a new line of therapy or disease subset may have multiple prior approvals. In addition, approvals of drugs as single agents or in combination may also have prior approval dates. For additional information regarding prior approvals, please refer to the downloadable “Pediatric Approvals Additional information” file and Drugs@FDA.

# 02-21-2023: label updated to Physician Labeling Rule (PLR) format; indication statement updated to specify “adults and pediatric patients 1 year of age and older” and remove the FAB classification

The Center for Drug Evaluation and Research generally divides the pediatric population into the following groups:

  • Neonates: birth up to 28 days (corrected gestational age)
  • Infants: 28 days up to 2 years
  • Children: 2 years up to 12 years
  • Adolescents: 12 years up to younger than 17 years

Further Information

 

 

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