On May 8, 2020, the FDA granted accelerated approval to selpercatinib for (i) 
adult patients with metastatic RET fusion-positive non-small cell lung cancer 
(NSCLC), (ii) adult and pediatric patients ≥12 years of age with advanced or 
metastatic RET-mutant medullary thyroid cancer who require systemic therapy, and 
(iii) adult and pediatric patients ≥12 years of age with advanced or metastatic 
RET fusion-positive thyroid cancer who require systemic therapy and who are 
radioactive iodine refractory (if radioactive iodine is appropriate). Approval 
was granted on the basis of the clinically important effects on the overall 
response rate (ORR) with prolonged duration of responses observed in a 
multicenter, open-label, multicohort clinical trial (LIBRETTO-001, NCT03157128) 
in patients whose tumors had RET alterations. ORRs within the approved patient 
populations ranged from 64% [95% confidence interval (CI), 54-73] in prior 
platinum-treated RET fusion-positive NSCLC to 100% (95% CI, 63-100) in systemic 
therapy-naïve RET fusion-positive thyroid cancer, with the majority of 
responders across indications demonstrating responses of at least 6 months. The 
product label includes warnings and precautions for hepatotoxicity, 
hypertension, QT interval prolongation, hemorrhagic events, hypersensitivity, 
risk of impaired wound healing, and embryo-fetal toxicity. This is the first 
approval of a drug specifically for patients with RET alterations globally.