NEW YORK – The US Food and Drug Administration on Friday granted full approval to Eli Lilly's Retevmo (selpercatinib) as a treatment for certain patients with advanced RET-mutated medullary thyroid cancer.
The FDA previously granted accelerated approval to the RET inhibitor in this patient population, first in patients older than age 12 in 2020, then in patients older than age 2 in May 2024.
Now, the agency is converting the previous accelerated approval to a full approval based on results from the Phase III LIBRETTO-531 trial, in which patients were randomized to receive either Retevmo or physician's choice of Exelixis' multi-kinase inhibitor Cabometyx (cabozantinib) or Sanofi's RTK inhibitor Caprelsa (vandetanib).
The median progression-free survival was not reached among patients treated with Retevmo at the time of data analysis versus 16.8 months among patients treated with Cabometyx or Caprelsa. According to a prespecified analysis of patient-reported side effects, patients in the Retevmo arm reported less time with severe side effects than those receiving Cabometyx or Caprelsa.