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# FDA OKs Retevmo for Pediatric Thyroid Cancer
- URL: https://www.fdaweb.com/fda-oks-retevmo-for-pediatric-thyroid-cancer/
- Published: 2024-05-29T12:00:00.000Z
- Updated: 2026-09-14T14:32:00.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5157080

FDA has granted Eli Lilly accelerated approval for Retevmo (selpercatinib) for pediatric patients two years of age and older with advanced or metastatic medullary thyroid cancer with a RET mutation, as detected by an FDA-approved test, who require systemic therapy. The therapy was also granted accelerated approval for advanced or metastatic thyroid cancer with a RET gene fusion, and who are radioactive iodine-refractory.

An [FDA release](https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-selpercatinib-pediatric-patients-two-years-and-older-ret-altered?ref=fdaweb.com) says this is the first FDA approval of a targeted therapy for pediatric patients less than 12 years of age with RET alterations. Selpercatinib was previously [granted](https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-selpercatinib-lung-and-thyroid-cancers-ret-gene-mutations-or-fusions?ref=fdaweb.com) accelerated approval for the thyroid cancer indications in adults and pediatric patients 12 years of age and older. It also was previously [granted](https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-selpercatinib-locally-advanced-or-metastatic-ret-fusion-positive-solid-tumors?ref=fdaweb.com) accelerated approval for the solid tumor indication in adults.

The recent approval was based on data from the LIBRETTO-121 (NCT03899792) single-arm, multi-cohort trial. FDA says the major efficacy outcome measures were confirmed overall response rate and duration of response, which were 48% and 92%, respectively.

The most common adverse reactions (seen in more than 25% of patients) were musculoskeletal pain, diarrhea, headache, nausea, vomiting, coronavirus infection, abdominal pain, fatigue, pyrexia, and hemorrhage, FDA says. The most common Grade 3 or 4 laboratory abnormalities were decreased calcium, decreased hemoglobin, and decreased neutrophils.