Table 1.

Efficacy of Selpercatinib in LIBRETTO-001

Prior platinum doublet (n = 105)
ORR (%)	68 (95% CI 58–76)
Medium DOR (months)	20.3 (95% CI 13.8–24.0)
Medium PFS (months)	18.4 (95% CI 12.9–24.9)
Treatment naïve (n = 34)
ORR (%)	85 (95% CI 69–95)
Medium DOR (months)	NR
PFS (months)	NR
Patients with measurable CNS metastasis (n = 11)
ORR (%)	91% (95% CI 59–100)

Most AEs were low grade. The most common adverse events of grade 3 or higher were hypertension (in 14% of the patients), an increased alanine aminotransferase level (in 12%), an increased aspartate aminotransferase level (in 10%), hyponatremia (in 6%), and lymphopenia (in 6%). Only 2% of patients discontinued selpercatinib due to AEs

NR, Not Reached; ORR, Objective Response Rates; DOR, Duration of Response; PFS, Progression-Free Survival