New Drug: Selpercatinib in RET-mutant Medullary Thyroid Cancer


  • Study

    Phase 3, randomized trial (LIBRETTO-531)
    RET mutant, unresectable, locally advanced or metastatic medullary thyroid cancer and no history of treatment with kinase inhibitors
    Selpercatinib (n=193) vs. SOC (Cabozantinib; Vandetanib)




  • Efficacy

    mPFS: NR vs. 16.8 mos, HR: 0.28, p<0.001
    12 mos PFS: 86.8% vs. 65.7%
    Median treatment failure free survival: NR vs. 13.9 mos, HR:0.25, p<0.001
    ORR: 69.4% vs. 39.8%
    CR: 11.9% vs. 4.1 %




  • Safety

    Grade3: Hypertension (18.7% vs. 17.5%), ALT increased (10.4% vs. 2.1%), diarrhea (3.1% vs. 8.2%), mucosal inflammation (0.5% ve 13.4%), palmar–plantar erythrodysesthesia syndrome (0 vs. 9.3%)



  • N Engl J Med 2023; 389:1851-186

    Phase 3 Trial of Selpercatinib in Advanced RET-Mutant Medullary Thyroid Cancer

    http://doi.org/10.1056/NEJMoa2309719

    Reviewed by Elvin Chalabiyev, MD on Nov 20, 2023