Summary
- On September 27, 2024, the FDA approved selpercatinib (Retevmo) for advanced or metastatic medullary thyroid cancer patients with a RET mutation.
- Selpercatinib was initially approved for patients 12 years and older in 2020, and later for those 2 years and older in 2024.
- The approval was based on a study showing selpercatinib’s effectiveness in prolonging progression-free survival compared to other treatments.
- Common side effects of selpercatinib include hypertension, edema, dry mouth, fatigue, and diarrhea.
- Healthcare professionals are encouraged to report any adverse events associated with selpercatinib to the FDA.
In a recent development, the Food and Drug Administration (FDA) has granted full approval to selpercatinib (Retevmo, Eli Lilly and Company) for the treatment of advanced or metastatic medullary thyroid cancer (MTC) in both adult and pediatric patients aged 2 years and older with a specific genetic mutation called RET. This approval expands the indication for selpercatinib, which was previously granted accelerated approval for patients 12 years and older in 2020 and received further accelerated approval for pediatric patients aged 2 years and older in May 2024.
The efficacy of selpercatinib was established through the LIBRETTO-531 study, showing a significant improvement in progression-free survival compared to standard treatments like cabozantinib or vandetanib. Some common side effects observed in patients taking selpercatinib include hypertension, edema, dry mouth, fatigue, and diarrhea. Healthcare professionals are encouraged to report any serious adverse events related to the medication to the FDA’s MedWatch Reporting System. This milestone in cancer treatment highlights the importance of expedited programs in bringing innovative therapies to patients in need.