FDA Approves Selpercatinib for Adult and Pediatric Patients With RET Fusion-Positive Thyroid Cancer
On June 12, 2024, the US Food and Drug Administration (FDA) granted traditional approval to selpercatinib for adult and pediatric patients 2 years and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and are radioactive iodine-refractory. This approval was based on results from the phase 1/2 LIBRETTO-001 study and supportive evidence from the phase 1/2 LIBRETTO–121 study.
In the open-label, multi-cohort LIBRETTO-001 study, 65 adult patients who received prior lines of systemic therapy (n = 41) or were systemic therapy-naive (n = 24) were enrolled in separate cohorts to receive selpercatinib. In the single-arm, multi-cohort LIBRETTO-121 study, 10 pediatric and young adult patients received selpercatinib. The major efficacy outcomes included overall response rate (ORR) and duration of response (DOR).
In the LIBRETTO-001 study, ORR was 85% in previously treated patients and 96% in treatment-naive patients. Median DOR was 26.7 months in previously treated patients and not evaluable in treatment-naive patients. In the LIBRETTO-121 study, ORR was 60% and among 83% of patients, the DOR was ≥ 12 months.
The most common adverse reactions occurring in ≥25% of patients included edema, diarrhea, fatigue, dry mouth, hypertension, abdominal pain, constipation, rash, nausea, and headache. The most common grade 3/4 laboratory abnormalities occurring in ≥5% of patients included decreased lymphocytes, increased alanine aminotransferase, increased aspartate aminotransferase, decreased sodium, and decreased calcium.
The recommended dose of selpercatinib is based on body surface area for patients aged 2 to 12. The recommended dose for patients aged 12 years and older is based on body weight.
Source:
FDA approves selpercatinib for RET fusion-positive thyroid cancer. Published online: June 12, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-selpercatinib-ret-fusion-positive-thyroid-cancer
