First RET Inhibitor OK’d for Kids

The FDA granted accelerated approval to selpercatinib (Retevmo; Eli Lilly) for children age 2 and older with advanced or metastatic medullary thyroid cancer with a RET mutation; advanced or metastatic or thyroid cancer with a RET fusion who are refractory to radioactive iodine; or locally advanced or metastatic solid tumors with a RET fusion that have advanced despite systemic therapy or for which there are no alternatives. Today's decision was based on the results of the 25-patient LIBRETTO-121 study, in which the overall response rate was 48% and the median duration of response was not reached, with disease control continuing beyond 12 months in 92% of patients. This is the first time the FDA has approved a RET inhibitor for children younger than 12.