The FDA granted regular approval to selpercatinib for treatment of certain patients with non-small cell lung cancer (NSCLC).

The indication applies to use of selpercatinib (Retevmo, Eli Lilly) by adults with locally advanced or metastatic RET fusion-positive NSCLC as detected by an FDA-approved test.

The multicenter, open-label LIBRETTO-001 trial evaluated selpercatinib — a kinase inhibitor — for 316 patients (median age, 61 years; range, 23-92; 58% female; 49% white, 41% Asian, 5% Black ) with locally advanced or metastatic RET fusion-positive NSCLC. Most patients (97%) had ECOG performance status of 0 or 1.

Study participants had received a median two (range, 1-15) prior systemic therapies, and more than half (58%) had received prior anti-PD-1/PD-L1 therapy.

Patients received selpercatinib dosed at 160 mg orally twice daily until disease progression or unacceptable toxicity. Major efficacy outcomes included overall response rate and duration of response as assessed by blinded independent review committee.

The FDA granted accelerated approval to selpercatinib for this indication in May 2020 based on initial overall response rate and duration of response data from 144 patients.

The agency converted the accelerated approval to regular approval based on data from the additional 172 patients, as well as an additional 18 months of follow-up that allowed investigators to assess response durability.