On July 14, 2026, the FDA granted traditional approval to selpercatinib (Retevmo) for adult and pediatric patients 2 years of age and older with locally advanced or metastatic solid tumors harboring a RET gene fusion, as detected by an FDA-approved test, after progression on prior systemic therapy or when no satisfactory alternative treatment options are available. The drug had previously received accelerated approval for this indication in adults in 2022 and in pediatric patients in 2024.
The approval was supported by data from the multicenter, open-label LIBRETTO-001 trial, which evaluated 75 patients with RET fusion-positive tumors other than non–small cell lung cancer and thyroid cancer. The overall response rate was 47%, with a median duration of response of 24.5 months. Responses were seen across multiple tumor types, including colorectal, pancreatic, salivary, soft tissue sarcoma, cholangiocarcinoma, breast, ovarian, and neuroendocrine cancers. Additional support came from pediatric and young adult data in LIBRETTO-121, where responses were observed in patients with congenital infantile fibrosarcoma, spindle cell sarcoma, and RET fusion-positive thyroid cancer.
The prescribing information includes warnings and precautions for hepatotoxicity, interstitial lung disease/pneumonitis, hypertension, QT prolongation, hemorrhagic events, hypersensitivity, tumor lysis syndrome, impaired wound healing, hypothyroidism, embryo-fetal toxicity, and slipped capital femoral epiphysis/slipped upper femoral epiphysis in pediatric patients.
Source
- US Food and Drug Administration. FDA grants traditional approval to selpercatinib for locally advanced or metastatic RET fusion-positive solid tumors. Accessed July 20, 2026. www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-traditional-approval-selpercatinib-locally-advanced-or-metastatic-ret-fusion-positiver