FDA Grants Traditional Approval to Retevmo for RET Fusion-Positive Solid Tumors

The FDA granted traditional approval to selpercatinib (Retevmo, Eli Lilly and Co) for adults and children aged 2 years and older with locally advanced or metastatic solid tumors harboring a RET gene fusion. Eligible patients must have a RET gene fusion detected by an FDA-approved test and must have experienced disease progression during or after prior systemic treatment or have no satisfactory alternative treatment options. Selpercatinib previously received accelerated approval for this indication in adults in 2022 and in pediatric patients aged 2 years and older in 2024.

Efficacy was evaluated in LIBRETTO-001, a multicenter, open-label, multicohort trial, with additional support from findings in RET fusion-positive non–small cell lung cancer and thyroid cancer. The evaluation included 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 observed across several tumor types, including colorectal, pancreatic adenocarcinoma, salivary, soft tissue sarcoma, cholangiocarcinoma, carcinoma of the skin, unknown primary, breast, bronchial carcinoid, ovarian, small intestine, and neuroendocrine (including pancreatic).

Pediatric and young adult efficacy was evaluated in LIBRETTO-121, another multicenter, open-label, multicohort trial. Participants had locally advanced, refractory RET fusion-positive solid tumors and were unresponsive to available therapies or had no standard systemic curative treatment available. Responses were observed in patients with RET fusion-positive thyroid cancer and in individual patients with congenital infantile fibrosarcoma and spindle cell sarcoma.

For adults and adolescents aged 12 years and older, the recommended dose is 120mg orally twice daily for patients weighing less than 50kg and 160mg twice daily for those weighing 50kg or more. The prescribing information provides separate dosing recommendations for children aged 2 to younger than 12 years.

Source: U.S. Food and Drug Administration. FDA grants traditional approval to selpercatinib for locally advanced or metastatic RET fusion-positive solid tumors. Content current as of July 14, 2026. Accessed July 16, 2026.