Eli Lilly’s Retevmo (selpercatinib) has been granted accelerated approval by the US Food and Drug Administration (FDA) for adult patients with locally advanced or metastatic solid tumours with a rearranged during transfection (RET) gene fusion that have progressed on or following prior systemic treatment or who have no satisfactory alternative treatment options, the company announced.
In addition to the tumour-agnostic approval, the FDA has simultaneously granted traditional approval for Retevmo in adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with a RET gene fusion, as detected by an FDA-approved test.
“Since its initial accelerated approval, Retevmo has shifted the treatment paradigm for patients with RET-altered cancers,” said David Hyman, chief medical officer, Loxo@Lilly.
“Retevmo is the first and only RET inhibitor to receive both tumour-agnostic accelerated approval and traditional approval in NSCLC, further supporting its ability to deliver meaningful clinical benefit for patients across diverse tumour types,” Hyman added.
The two approvals are supported by data from the pivotal LIBRETTO-001 trial, which is the largest clinical trial of patients with RET-driven cancers treated with a RET inhibitor, the company outlined.
The multicentre, open-label, multi-group study enrolled with locally advanced or metastatic RET-driven solid tumours, including NSCLC.
Among the 41 patients in the tumour-agnostic data set, an overall response rate (ORR) of 44% was achieved. Complete responses (CRs) were seen in 4.9% of patients, partial responses (PRs) in 39% of patients, and the median duration of response (DoR) was 24.5 months.
In the treatment-naïve RET fusion-positive NSCLC population, ORR was 84%, with CRs in 5.8% of patients, and PRs observed in 78% of patients. The median DOR was 20.2 months, with responses ongoing in 40% of the treatment-naïve population at the time of data cutoff.
The ORR was 61% in chemotherapy-pretreated RET fusion-positive NSCLC population, with CRs in 7.3% of patients. PRs were seen in 54% of patients, and the median DOR in the pretreated group was 28.6 months.
Vivek Subbiah, associate professor of investigational cancer therapeutics at The University of Texas MD Anderson Cancer Center and co-investigator for LIBRETTO-001, said: “In the LIBRETTO-001 trial, [Retevmo] demonstrated clinically meaningful and durable responses across a variety of tumour types in patients with RET-driven cancers, including pancreatic, colon and other cancers in need of new treatment options.”