This list below includes molecular targets for which there is evidence and/or biologic rationale (gene expression data, genetic alterations, non-clinical or clinical data obtained from publicly available databases and/or peer-reviewed publications) to suggest their potential relevance to the growth or progression of one or more pediatric cancers.
The list fulfills the statutory requirements imposed by FDARA and is expected to provide some guidance to industry in planning for initial Pediatric Study Plan submissions for new drug and/or biologic products in development for cancer in accordance with the amended provisions of the Pediatric Research Equity Act. The absence of a molecular target to which a specific drug is directed from the Relevant Molecular Target List does not mean a clinical evaluation of the drug in the pediatric population will not be required. As well, the presence of a target on the Relevant Molecular Target List in itself does not automatically constitute a requirement for a clinical study.
The list will be regularly updated as new information becomes available.