FDA grants full approval to selpercatinib for RET+ thyroid cancer – Eli Lilly

The FDA has granted full approval to selpercatinib (Retevmo) from Eli Lilly for the treatment of adult and pediatric patients at least 2 years of age with advanced or metastatic RET fusion–positive thyroid cancer who require systemic therapy and who are radioactive iodine (RAI) refractory, if RAI is appropriate. Selpercatinib received accelerated approval in the same indication in May 2020.

The FDA’s full approval was supported by data from the phase 1/II LIBRETTO-001 trial (NCT03157128), which showed that patients who received prior treatment (n = 41) achieved an overall response rate (ORR) of 85% (95% CI, 71%-94%), and patients who were naive to systemic therapy (n = 24) experienced an ORR of 96% (95% CI, 79%-100%). Previously treated patients had a median duration of response (DOR) of 26.7 months (95% CI, 12.1–not evaluable [NE]), and systemic therapy–naive patients had a median DOR that was NE (95% CI, 42.8-NE).

The full approval was also supported by efficacy data from 10 pediatric and young adult patients with thyroid cancer harboring RET fusions who were treated with selpercatinib during the phase 1/II LIBRETTO-121 trial (NCT03899792), which evaluated the agent in pediatric and young adult patients with advanced RET-altered solid tumors. These 10 patients experienced an ORR of 60% (95% CI, 26%-88%), and 83% of responders experienced a DOR of at least 12 months.