FDA Approves Selpercatinib for RET Fusion+ Medullary Thyroid Cancer

Support for the regulatory decision came from the multicenter, open-label LIBRETTO-531 trial (NCT04211337).

The FDA granted traditional approval to selpercatinib (Retevmo) for the treatment of adult and pediatric patients at least 2 years old with advanced or metastatic medullary thyroid cancer with a RET mutation, as detected by an FDA-approved test, who require systemic therapy.¹

Support for the regulatory decision came from the multicenter, open-label LIBRETTO-531 trial (NCT04211337), which showed that the median progression-free survival was not reached (NR; 95% CI, NE) in patients who were randomly assigned to selpercatinib vs 16.8 months (95% CI, 12.2-25.1) for those who were treated with cabozantinib (Cabometyx) or vandetanib (Caprelsa; HR, 0.280; 95% CI, 0.165-0.475; P <0.0001).

The FDA initially granted accelerated approval to selpercatinib for this indication in patients aged at least 12 years old in 2020. An accelerated approval for the agent was granted in May 2024 to pediatric patients aged 2 years and older.

Reference

1. FDA approves selpercatinib for RET fusion-positive medullary thyroid cancer. FDA. News release. September 27, 2024. Accessed September 27, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-selpercatinib-ret-fusion-positive-medullary-thyroid-cancer