Promising updates regarding a novel drug, selpercatinib (Retevmo®) for medullary thyroid cancer (MTC), have just been released, according to a press release by Eli Lilly Company, the maker of the drug.

“These data from the LIBRETTO-531 trial confirm the importance of selectivity in targeting RET-driven cancer,” said David Hyman, MD, chief medical officer of Loxo@Lilly.

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This research assessed the efficacy of selpercatinib as an initial treatment option for patients afflicted with advanced or metastatic rearranged during transfection (RET)-mutant MTC compared to other multikinase inhibitors, such as cabozantinib or vandetanib. The results showed that selpercatinib yielded a statistically significant and clinically substantial advancement in progression-free survival (PFS), which was the study’s primary goal.

The LIBRETTO-531 trial, a phase 3, randomized, open-label investigation, involved 291 patients. The investigators randomized patients into 2 groups: 1 receiving selpercatinib and the other receiving cabozantinib or vandetanib. The study evaluated the performance of selpercatinib, a highly selective and potent RET kinase inhibitor. Notably, it demonstrated better results in terms of PFS compared to the other multikinase inhibitors.

“Taken together with the recent positive selpercatinib phase 3 LIBRETTO-431 announcement in lung cancer, these results underscore the importance of timely and broad-based genomic testing to ensure patients who could potentially benefit receive targeted therapies,” added Dr. Hyman.

The study also revealed that adverse events observed with selpercatinib were generally consistent with those identified in the drug’s earlier development programs. The medication’s label contains warnings and precautions for various potential side effects, including hemorrhagic events, interstitial lung disease, hepatotoxicity, QT interval prolongation, pneumonitis, hypersensitivity, hypertension, tumor lysis syndrome, risk of impaired wound healing, embryo-fetal toxicity, and hypothyroidism.

Medullary thyroid cancer, which accounts for a small percentage of thyroid cancers, is characterized by RET mutations. These mutations are present in most cases, emphasizing the importance of effective treatments like selpercatinib.


Lilly’s Retevmo® (selpercatinib) demonstrates superior progression-free survival compared to approved multikinase inhibitors in ret-mutant medullary thyroid cancer. Lilly; August 22, 2023.