Key takeaways:

• The FDA approved selpercatinib for patients aged 2 years and older with certain solid tumors.
• Data from an open-label, multicohort trial showed a median duration of response of more than 2 years.

The FDA has granted traditional approval to selpercatinib for adults and children aged 2 years and older with locally advanced or metastatic solid tumors with a RET gene fusion.

Selpercatinib (Retevmo, Eli Lilly & Co.), an oral kinase inhibitor, received approval for treatment of patients with RET fusion-positive tumors, as detected by an FDA-approved test, who have progressed on or following systemic therapy or who have no alternative treatment options.

The FDA has granted traditional approval to selpercatinib for adults and children aged 2 years and older with locally advanced or metastatic solid tumors with a RET gene fusion.

The agent previously received accelerated approval for this indication in 2022 for adults and in 2024 for pediatric patients aged 2 years and older, according to an FDA release.

The FDA based its decision on data from the open-label LIBRETTO-001 trial, which included 75 patients with RET fusion-positive tumors, other than non-small cell lung cancer and thyroid cancer.

Eligible patients had experienced disease progression on or following previous systemic therapy or had no alternative treatment options.

In LIBRETTO-001, researchers observed an overall response rate of 47% (95% CI, 35%-59%) and a median duration of response of 24.5 months (95% CI, 11.2-49.1).

The FDA also evaluated data from the LIBRETTO-121 trial, which included pediatric and young adult patients with locally advanced refractory RET fusion-positive solid tumors who had not responded to prior therapies and had no standard systemic curative therapy option.

Researchers observed responses in one patient with congenital infantile fibrosarcoma and another with a spindle cell sarcoma, as well as in patients with RET fusion-positive thyroid cancer.

Prescribing information for selpercatinib for warnings and precautions for hepatotoxicity, interstitial lung disease/pneumonitis, hypertension, QT interval prolongation, hemorrhagic events, hypersensitivity, tumor lysis syndrome, risk for impaired wound healing, hypothyroidism, embryo-fetal toxicity and slipped capital femoral epiphysis/slipped upper femoral epiphysis in children.