NEW YORK – The US Food and Drug Administration on Wednesday granted full approval to Eli Lilly's Retevmo (selpercatinib) as a treatment for patients with advanced or metastatic RET fusion-positive thyroid cancer.
The RET inhibitor is indicated for patients who require systemic therapy and whose cancers are refractory to radioactive iodine.
The latest FDA action converts Retevmo's 2020 accelerated approval from the agency in advanced RET fusion-positive thyroid cancer to a full approval. While the accelerated approval made the drug available to patients who were at least 12 years old, now doctors can prescribe the drug to patients who are as young as 2 years old.
The agency approved the drug after reviewing results from the Phase I/II LIBRETTO-001 trial, which included 65 patients, some of whom had received prior systemic therapy and some of whom hadn't. Among 41 previously treated patients, the overall response rate was 85 percent, and the median duration of response was 26.7 months. Among 24 patients who hadn't gotten systemic therapy before, the overall response rate was 96 percent, and the median duration of response had not been reached at the time of data cutoff.
The FDA also considered supporting evidence from the Phase I/II LIBRETTO-121 trial, which included pediatric and young adult patients with advanced RET-altered solid tumors. In that trial, the overall response rate was 60 percent, and 83 percent had a duration of response that was at least 12 months.
Retevmo is also FDA-approved for patients with advanced or metastatic RET fusion-positive non-small cell lung cancer; for advanced or metastatic medullary thyroid cancers harboring RET mutations; and for RET fusion-positive solid tumor patients whose cancers are refractory to prior treatment and do not have other treatment options. In 2022, the agency approved Thermo Fisher Scientific's next-generation sequencing assay, the Oncomine DX Target Test, as a companion diagnostic to identify patients with advanced RET-driven lung and thyroid cancers who may be eligible for Retevmo. The following year, the FDA also approved Foundation Medicine's FoundationOne CDx as a companion diagnostic for Retevmo.