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Selpercatinib
Source: Childhood Multiple Endocrine Neoplasia (MEN) Syndromes Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: April 3, 2025 · Captured 2026-09-09.
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Selpercatinib is a RET inhibitor.
A phase I/II trial of selpercatinib therapy included patients with cancers and RET variants. The study enrolled 55 patients with medullary thyroid cancer (age range, 17–84 years) who were previously treated with vandetanib and/or cabozantinib and 88 patients with medullary thyroid cancer (age range, 15–82 years) who were not previously treated with vandetanib or cabozantinib.[25]
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For the previously treated cohort, 69% of patients achieved objective responses, and the median duration of response had not been reached, with a median follow-up of 14 months.
For the cohort who were not previously treated, 73% of patients achieved objective responses, with a median duration of response of 22.0 months.
The most common grades 3 to 4 treatment-related adverse events were hypertension (12%), increased alanine aminotransferase (10%) and aspartate aminotransferase (7%), diarrhea (3%), and prolonged QT interval (2%).
The U.S. Food and Drug Administration granted traditional approval to selpercatinib for the treatment of adult and pediatric patients aged 2 years and older with advanced or metastatic RET-variant medullary thyroid cancer who require systemic therapy.[26]
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