Eli Lilly and Company announced results from both the LIBRETTO-431 Phase 3 study, which evaluated Retevmo (selpercatinib) versus platinum-based chemotherapy — with or without pembrolizumab — as an initial treatment for patients with advanced or metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC), and the LIBRETTO-531 Phase 3 study, which evaluated Retevmo versus multikinase inhibitors (MKIs) in patients with advanced or metastatic RET-mutant medullary thyroid cancer (MTC). In both clinical studies, results were based on pre-specified interim efficacy analyses conducted by independent data monitoring committees (IDMC). Results from the LIBRETTO-431 and LIBRETTO-531 Phase 3 trials were presented in a Presidential Symposium at the European Society for Medical Oncology (ESMO) Congress 2023 and simultaneously published in the New England Journal of Medicine.

"These results from LIBRETTO-431 and LIBRETTO-531 are striking and provide critical evidence supporting the importance of optimizing initial therapy for patients with RET-driven cancers," said David Hyman, M.D., chief medical officer, Lilly. "We are excited to be sharing these data with the clinical community in both NEJM and at ESMO. It is our hope that these data lead to further adoption of biomarker testing in the initial treatment journey for people with NSCLC and MTC and help make Retevmo a standard initial treatment option for all appropriate patients."

The labeling for Retevmo contains warnings and precautions for hepatotoxicity (evidence of liver dysfunction), interstitial lung disease (ILD)/pneumonitis, hypertension, QT interval prolongation, hemorrhagic events, hypersensitivity, tumor lysis syndrome, risk of impaired wound healing, hypothyroidism, and embryo-fetal toxicity.