FDA approves selpercatinib for RET fusion-positive thyroid cancer
Regulatory approval: On June 12, 2024, the FDA granted traditional approval to selpercatinib (Retevmo) for adults and pediatric patients aged ≥2 years with advanced or metastatic RET fusion–positive thyroid cancer requiring systemic therapy and who are radioactive iodine (RAI)-refractory (if RAI is appropriate). Efficacy was primarily supported by LIBRETTO-001, reporting high overall response rates in both previously treated and systemic therapy–naïve cohorts; supportive pediatric/young adult data came from LIBRETTO-121. Common adverse reactions and key grade 3/4 lab abnormalities were reported.
FDA approval (regulatory approval)
On June 12, 2024, the FDA granted traditional approval to selpercatinib (Retevmo; Eli Lilly and Company) for adult and pediatric patients ≥2 years with advanced or metastatic RET fusion–positive thyroid cancer who require systemic therapy and are radioactive iodine (RAI)-refractory (if RAI is appropriate). Selpercatinib previously received accelerated approval for this indication in 2020 for patients ≥12 years.
Supporting evidence
LIBRETTO-001 (NCT03157128), a multicenter, open-label, multi-cohort trial, evaluated 65 patients with RET fusion–positive thyroid cancer who were RAI-refractory (if applicable), including separate cohorts of systemic therapy–naïve and previously treated patients. Primary outcomes were ORR and DOR:
- Previously treated (n=41): ORR 85% (95% CI, 71–94); median DOR 26.7 months (95% CI, 12.1–NE)
- Systemic therapy–naïve (n=24): ORR 96% (95% CI, 79–100); median DOR NE (95% CI, 42.8–NE)
Supportive evidence included LIBRETTO-121 (NCT03899792) in 10 pediatric/young adult patients: ORR 60% (95% CI, 26–88); 83% had observed DOR ≥12 months.
Safety (selected)
Common adverse reactions (≥25%): edema, diarrhea, fatigue, dry mouth, hypertension, abdominal pain, constipation, rash, nausea, headache. Common grade 3/4 lab abnormalities (≥5%): decreased lymphocytes, increased ALT/AST, decreased sodium, decreased calcium.