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Since 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 June 2022. Updates will be made as new information becomes available.

Downloadable file for: Pediatric Approvals Additional Information

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 up to 12 years
• Adolescents: 12 years up to 16 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