Selpercatinib

Indication. Selpercatinib is indicated for adult patients with locally advanced or metastatic solid tumors with a RET gene fusion whose disease meets either of the following criteria: disease progressed during or after prior systemic treatment, or disease with no satisfactory alternative treatment options. This indication is not approved for the pediatric population.

Evidence. Forty-one patients with RET gene fusion–positive tumors (other than non-small cell lung cancer [NSCLC] and thyroid cancer) with disease progression during or after prior systemic treatment or who had no satisfactory alternative treatment options were evaluated in the multicenter, open-label, multicohort LIBRETTO-001 trial (NCT03157128).[1,2][Level of evidence C3]

Efficacy. For the 41 evaluable patients with cancers other than NSCLC or thyroid cancer, the objective response rate was 44% (95% confidence interval, 28%–60%). A total of 16 patients had a partial response and 2 patients had a complete response, with a median follow-up of 14.9 months.[1,2]

Identification of biomarker. Clinical Laboratory Improvement Amendments (CLIA) or CLIA-equivalent locally obtained molecular testing was performed at a certified laboratory, with the use of either next-generation sequencing, fluorescence in situ hybridization, or polymerase chain reaction assay.