Selpercatinib Granted Full Approval

The RET inhibitor received traditional approval to treat RET-fusion-positive solid tumors.

The U.S. Food and Drug Administration (FDA) has granted full approval to selpercatinib (Retevmo) to treat RET-fusion-positive solid tumors in adult and pediatric patients (2 years and older). This indication is for patients whose RET gene fusion has been confirmed by an FDA-approved test and whose disease has progressed following prior systemic treatment, with no alternative therapeutic options available.

Selpercatinib is a targeted therapy that inhibits RET, a protein that helps regulate cell growth. RET gene fusions occur when part of the RET gene joins another gene, producing abnormal proteins that can drive cancer growth.

Person pouring medication out of pill bottle into hand.

Selpercatinib previously received accelerated approval for adult patients in September 2021 and for pediatric patients in May 2024. Conversion to traditional approval was based on confirmatory evidence from the LIBRETTO-001 and LIBRETTO-121 trials. The approval adds to the growing number of biomarker-driven, tumor-agnostic cancer therapies that treat cancers based on their molecular alterations rather than their tissue of origin.

LIBRETTO-001 is a multicenter, open-label, multicohort phase I/II clinical trial. The clinical trial enrolled 75 patients with RET-fusion positive tumors other than non-small cell lung cancer (NSCLC) and thyroid cancer who experienced disease progression during or after prior systemic therapy or had no satisfactory alternative treatment options.

Investigators found that 47% of patients had responses to selpercatinib treatment, with responses lasting a median of 24.5 months. Responses were observed across a range of tumor types, including colorectal cancer, pancreatic adenocarcinoma, salivary gland tumors, soft tissue sarcoma, cholangiocarcinoma, skin carcinoma, cancer of unknown primary, breast cancer, bronchial carcinoid, ovarian cancer, small intestinal cancer, and neuroendocrine tumors (including pancreatic neuroendocrine tumors). Additional supporting evidence came from patients in other LIBRETTO-001 cohorts, including patients with RET-fusion positive NSCLC and RET-fusion positive thyroid cancer.

LIBRETTO-121 is a multicenter, open-label, multicohort phase I/II clinical trial that evaluated selpercatinib in pediatric and young adult patients with locally advanced, refractory, RET fusion-positive solid tumors who did not have responses to available therapies or had no standard systemic curative therapy available. Responses to selpercatinib were observed among patients with congenital infantile fibrosarcoma, spindle cell carcinoma, and thyroid cancer.

The recommended dose of selpercatinib for adult and adolescent patients 12 years of age or older is based on body weight:

  • Patients weighing <50 kg: 120 mg orally twice daily
  • Patients weighing ≥50 kg: 160 mg orally twice daily

For pediatric patients aged 2 to younger than 12 years old, the recommended dosage is based on body surface area:

  • Body surface area of 0.33 m2 to 0.65 m2: 40 mg orally three times daily
  • Body surface area of 0.66 m2 to 1.08 m2: 80 mg orally twice daily
  • Body surface area of 1.09 m2 to 1.52 m2: 120 mg orally twice daily
  • Body surface area ≥1.53 m2: 160 m2 orally twice daily

RET gene fusions are estimated to occur in 1% to 2% of NSCLCs, approximately 20% of papillary thyroid cancers, and less than 1% of many other solid tumors.

The FDA rendered its decision on July 14, 2026. Check this resource for updated information on all therapeutics regulated by the FDA.