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                Oncology Center of Excellence

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          Pediatric Oncology Drug ApprovalsOncology Center of Excellence Pediatric Oncology Drug Approvals
  
  	
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              OCE Pediatric Drugs HeaderSince the early 1950s, the FDA has approved more than 50 drugs with over 70 indications for the treatment of pediatric cancers. From the older broad-spectrum cytotoxic chemotherapy drugs to the more recent molecularly-targeted drugs, the number of oncology products approved for use in children has been expanding as a result of recent changes in the regulatory framework to accelerate pediatric drug development and the increased understanding of tumor biology.

The approvals reflect the variety of strategies applied to pediatric drug development. Examples include pediatric-specific indications, indications based on supplemental approvals of drugs initially approved for adult indications, indications with extrapolation of adult efficacy data to pediatric populations, and tumor-agnostic indications.

Diverse therapeutic agents such as small-molecule kinase inhibitors, monoclonal antibodies, and other antibody-based therapies (e.g., bispecific antibody [BiTE], antibody-drug conjugate [ADC], 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 drug 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 March 2023. 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 3Dabrafenib [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
			03-16-2023Trametinib [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
			03-16-2023Nivolumab [Opdivo]
			Adjuvant treatment of adult and pediatric patients 12 years and older with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection
			02-15-2023Nivolumab [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
			02-15-2023Atezolizumab [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)
			12-09-2022Brentuximab 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
			11-10-2022Dabrafenib [Tafinlar]
			In combination with trametinib, for the treatment of adult and pediatric patients 6 years 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
			06-22-2022Trametinib [Mekinist]
			In combination with dabrafenib, for the treatment of adult and pediatric patients 6 years 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
			06-22-2022Azacitidine [Vidaza]
			Treatment of pediatric patients aged 1 month and older with newly diagnosed Juvenile Myelomonocytic Leukemia (JMML)
			05-20-2022Nivolumab and Relatlimab-rmbw) [Opdualag]
			Treatment of adult and pediatric patients 12 years of age or older with unresectable or metastatic melanoma
			03-18-2022Pembrolizumab [Keytruda]
			Adjuvant treatment of adult and pediatric (12 years and older) patients with Stage IIB, IIC, or III melanoma following complete resection
			12-03-2021Rituximab [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
			12-02-2021Cabozantinib [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
			09-17-2021Asparaginase 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
			06-30-2021Daunorubicin 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
			03-30-2021Crizotinib [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
			01-14-2021Crizotinib [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
			07-14-2022Pralsetinib [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
			12-01-2020Pralsetinib [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)
			12-01-2020Naxitamab-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
			11-25-2020Pembrolizumab [Keytruda]
			Treatment of pediatric patients with refractory classical Hodgkin lymphoma (cHL), or cHL that has relapsed after 2 or more lines of therapy
			10-14-2020Gemtuzumab ozogamicin [Mylotarg]
			Treatment of newly-diagnosed CD33-positive acute myeloid leukemia in adults and pediatric patients 1 month and older
			06-16-2020Pembrolizumab [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
			06-16-2020Selpercatinib [Retevmo]
			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
			05-08-2020Selpercatinib [Retevmo]
			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)
			05-08-2020Selumetinib [Koselugo]
			Treatment of pediatric patients 2 years of age and older with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN)
			04-10-2020Tazemetostat [Tazverik]
			Treatment of adults and pediatric patients aged 16 years and older with metastatic or locally advanced epithelioid sarcoma not eligible for complete resection
			01-23-2020Entrectinib [Rozlytrek]
			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 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 alternative therapy
			08-15-2019Dasatinib [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
			12-21-2018Tagraxofusp-erzs [Elzonris]
			Treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients 2 years and older
			12-21-2018Calaspargase 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
			12-20-2018Pembrolizumab [Keytruda]
			Treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC)
			12-19-2018Larotrectinib [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
			11-26-2018Emapalumab-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
			11-20-2018Iobenguane 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
			07-30-2018Ipilimumab [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
			07-10-2018Pembrolizumab [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
			06-13-2018Blinatumomab [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 adults and children
			03-29-2018Nilotinib [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
			03-22-2018Nilotinib [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
			03-22-2018Dasatinib [Sprycel]
			Treatment of pediatric patients 1 year of age and older with Philadelphia chromosome-positive (Ph+) chronic myeloid leukemia (CML) in chronic phase
			11-09-2017Gemtuzumab ozogamicin [Mylotarg]
			Treatment of relapsed or refractory CD33-positive acute myeloid leukemia in adults and pediatric patients 2 years and older.
			09-01-2017Tisagenlecleucel [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
			08-30-2017Nivolumab [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
			07-10-2018 **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 adults and children
			07-11-2017Pembrolizumab [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
			05-23-2017Avelumab [Bavencio]
			Treatment of adults and pediatric patients 12 years and older with metastatic Merkel cell carcinoma (MCC)
			03-23-2017Dinutuximab [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
			03-10-2015Denosumab [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
			06-13-2013Imatinib Mesylate [Gleevec]
			Pediatric patients with newly diagnosed Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL) in combination with chemotherapy
			01-25-2013Asparaginase 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
			11-18-2011Everolimus [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
			10-29-2010Imatinib Mesylate [Gleevec]
			Newly diagnosed adult and pediatric patients with Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase
			09-27-2006Pegaspargase [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)
			07-24-2006Nelarabine [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
			10-28-2005Clofarabine [Clolar]
			Treatment of pediatric patients 1 to 21 years old with relapsed or refractory acute lymphoblastic leukemia after at least two prior regimens
			12-28-2004Busulfan [Busulfex]
			For use in combination with cyclophosphamide as a conditioning regimen prior to allogeneic hematopoietic progenitor cell transplantation for chronic myelogenous leukemia
			01-13-2003Arsenic 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
			09-25-2000Tretinoin
			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
			02-01-1994Daunorubicin Hydrochloride
			In combination with other approved anticancer drugs, for remission induction in acute lymphocytic leukemia of children and adults
			12-19-1979Asparaginase [Elspar]
			As a component of a multi-agent chemotherapeutic regimen for the treatment of patients with acute lymphoblastic leukemia (ALL)
			01-10-1978Lomustine [Gleostine]
			Treatment of patients with primary and metastatic brain tumors following appropriate surgical and/or radiotherapeutic procedures
			08-04-1976Lomustine [Gleostine]
			As a component of combination chemotherapy for the treatment of patients with Hodgkin's lymphoma whose disease has progressed following initial chemotherapy
			08-04-1976Doxorubicin Hydrochloride
			MULTIPLE
			08-07-1974Procarbazine Hydrochloride [Matulane]
			In combination with other anticancer drugs for the treatment of Stage III and IV Hodgkin's disease
			07-22-1969Cytarabine
			In combination with other approved anti-cancer drugs, for remission induction in acute non-lymphocytic leukemia of adults and pediatric patients
			06-17-1969Thioguanine [Tabloid]
			Remission induction and remission consolidation treatment of acute nonlymphocytic leukemiasVinblastine sulfate
			Palliative treatment of malignancies including generalized Hodgkin’s disease, histiocytosis X and testicular germ cell carcinoma
			11-25-1965Dactinomycin [Cosmegen]
			Treatment of adult and pediatric patients with Wilms tumor, as part of a multi-phase, combination chemotherapy regimen
			12-10-1964Dactinomycin [Cosmegen]
			Treatment of adult and pediatric patients with rhabdomyosarcoma, as part of a multi-phase, combination chemotherapy regimen
			12-10-1964Dactinomycin [Cosmegen]
			Treatment of adult and pediatric patients with Ewing sarcoma, as part of a multi-phase, combination chemotherapy regimen
			12-10-1964Dactinomycin [Cosmegen]
			Treatment of adult and pediatric patients with metastatic, nonseminomatous testicular cancer, as part of a multi-phase, combination chemotherapy regimen
			12-10-1964Vincristine Sulfate
			Acute leukemia; MULTIPLE (in combination)Cyclophosphamide [Cytoxan]
			MULTIPLE
			11-16-1959Methotrexate
			Treatment of adult and pediatric patients with acute lymphoblastic leukemia (ALL) as part of a combination chemotherapy regimen
			12-07-1953Methotrexate
			Prophylaxis and treatment of meningeal leukemia in adult and pediatric patients
			12-07-1953Methotrexate
			Treatment of adults and pediatric patients with Non-Hodgkin lymphoma
			12-07-1953Methotrexate
			Treatment of adults and pediatric patients with osteosarcoma as part of a combination chemotherapy regimen
			12-07-1953Mercaptopurine [Purinethol tablet] &
			Treatment of adult and pediatric patients with acute lymphoblastic leukemia (ALL) as part of a combination chemotherapy maintenance regimen
			09-11-1953

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.
	Column B: [INDICATION]: This table reflects current data as of March 2023. To view additional information, please check its Drugs@FDA listing.
	Column C: [PEDIATRIC APPROVAL DATE]: Missing dates: Not available in digital records.* Everolimus (Afinitor disperz: 08-29-2012); & Mercaptopurine (Purixan suspension: 04-28-2014)
** 07-10-2018 (combination), 07-31-2017 (single agent)
*** 02-15-2023 (combination), 07-21-2017 (single agent)
# 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 1 month
	Infants: 1 month up to 2 years
	Children: 2 years up to 12 years
	Adolescents: 12 years up to younger than 17 years**Further Information**

FDA’s Labeling Resources for Human Prescription Drugs
	Prescribing Information Resources
	NCI: Drugs Approved for Childhood Cancers
	FDA Guidance: General Clinical Pharmacology Considerations for Pediatric Studies for Drugs and Biological Product
	Pediatric Oncology

                                                                                  Content current as of:
      03/31/2023Oncology Center of Excellence

          
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