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FDA Approves Selpercatinib for Adult and Pediatric Patients With Advanced or Metastatic Medullary Thyroid Cancer
On September 27, 2024, the US Food and Drug Administration (FDA) approved selpercatinib for adult and pediatric patients 2 years and older with advanced or metastatic medullary thyroid cancer harboring a RET mutation, as detected by an FDA-approved test. This conversion from accelerated to traditional approval was based on results from the phase 3 LIBRETTO-531 study.
In this open-label, multicenter study, adult and adolescent patients with advanced or metastatic medullary thyroid cancer who require systemic therapy were randomized on a 2-to-1 basis to receive selpercatinib (160 mg twice daily), or physician’s choice of either cabozantinib (140 mg daily) or vandetanib (300 mg daily). Randomization was stratified based on RET mutation status and intended treatment in the control arm. The primary end point was progression-free survival (PFS), as assessed by blinded independent review. A key secondary end point was safety.
At analysis, median PFS was not reached in the selpercatinib arm and was 16.8 months in the control arm (hazard ratio [HR] 0.28; 95% confidence interval [CI], 0.165 to 0.475; P < .0001). The most common adverse reactions occurring in ≥ 25% of patients included edema, dry mouth, fatigue, and diarrhea. The most common grade 3/4 laboratory abnormalities occurring in ≥ 5% of patients included decreased lymphocytes, increased alanine aminotransferase, decreased neutrophils, increased alkaline phosphatase, increased blood creatinine, decreased calcium, and increased aspartate aminotransferase. Patients in the selpercatinib arm reported less time with severe side effects than patients in the control arm.
The recommended dose for pediatric patients 12 years and under is based on body surface area. The recommended dose for patients 12 years and older is based on weight.
Source:
FDA approves selpercatinib for RET fusion-positive medullary thyroid cancer. Accessed on September 27, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-selpercatinib-ret-fusion-positive-medullary-thyroid-cancer
