Selpercatinib (Retevmo, Eli Lilly and Company) is now available for adult and pediatric patients 2 years of age and older with advanced or metastatic medullary thyroid cancer (MTC) with a RET mutation, as detected by an FDA-approved test, who require systemic therapy.1 Eli Lilly reported on their accelerated approval for this indication for patients 12 years of age and older in 2020, noting that it was based on data gathered in the LIBRETTO-531 (NCT04211337) clinical trial.2,3
In this multicenter, open-label study in adults and adolescents with advanced or metastatic RET-mutant MTC, 193 patients were randomly assigned in a 2-to-1 fashion to receive either a 160 mg twice daily dosing of selpercatinib or a once-daily physicians’ choice of 140 mg of cabozantinib or 300 mg of vandetanib. Patients were stratified based on RET mutation (M918T vs other) and intended treatment if the patients had been assigned to the control arm.
Median progression-free survival was not reached in the selpercatinib arm and was 16.8 months in the cabozantinib/vandetanib arm. The researchers noted that patients in the selpercatinib arm reported less time with severe adverse events, which included hypertension, edema, diarrhea, dry mouth, and fatigue. The most common grade 3 or 4 laboratory abnormalities were decreased lymphocytes, increased alanine aminotransferase, decreased neutrophils, increased alkaline phosphatase (ALP), increased blood creatinine, decreased calcium, and increased aspartate aminotransferase (AST).
On May 29, 2024, the FDA granted accelerated approval for this indication to pediatric patients 2 years of age and older, and on September 27, 2024, officials with the FDA reported this approval. This review used the assessment aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.
References
- U.S. Food and Drug Administration approves perioperative treatment of neoadjuvant Opdivo (nivolumab) and chemotherapy followed by surgery and adjuvant single-agent Opdivo for resectable non-small cell lung cancer (NSCLC). Press release. Bristol Myers Squibb. October 3, 2024. Accessed October 10, 2024. https://news.bms.com/news/corporate-financial/2024/U.S.-Food-and-Drug-Administration-Approves-Perioperative-Treatment-of-Neoadjuvant-Opdivo-nivolumab-and-Chemotherapy-Followed-by-Surgery-and-Adjuvant-Single-Agent-Opdivo-for-Resectable-Non-Small-Cell-Lung-Cancer-NSCLC/default.aspx
- FDA approves selpercatinib for RET fusion-positive medullary thyroid cancer. FDA. September 27, 2024. Accessed October 10, 2024. www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-selpercatinib-ret-fusion-positive-thyroid-cancer
- FDA approves selpercatinib for medullary thyroid cancer with a RET mutation. September 27, 2024. Accessed October 10, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-selpercatinib-medullary-thyroid-cancer-ret-mutation