Selpercatinib (Retevmo®), a drug developed by Eli Lilly and Company for the treatment of advanced solid RET-associated tumors such as medullary thyroid cancer (MTC), pancreatic cancer, and colon cancer, recently received US Food and Drug Administration approval, according to a press release. 

Selpercatinib is a selective RET kinase inhibitor. The data supporting its approval come from the results of the LIBRETTO-001 trial, which included patients from 16 countries and 85 health centers.

The primary outcome measures were the overall rate of response; secondary outcome measures included safety and duration of response. Results were evaluated by a blinded independent review committee.

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“In the LIBRETTO-001 trial, selpercatinib demonstrated clinically meaningful and durable responses across a variety of tumor types in patients with RET-driven cancers, including pancreatic, colon, and other cancers in need of new treatment options,” said Vivek Subbiah, MD, associate professor of investigational cancer therapeutics at the University of Texas MD Anderson Cancer Center and co-investigator for LIBRETTO-001.

The most common cancers included in the study were pancreatic adenocarcinoma (27%), colorectal (24%), salivary (10%), and unknown primary (7%). It is worth noting that over 90% of the included patients had previously received some form of systemic therapy before receiving selpercatinib. 

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The cohort had an overall response rate of approximately 45%, with an overall duration of response of approximately 25 months. A second cohort, including only patients with non-small cell lung cancer, had an overall response rate of 84%. The overall duration of treatment was a median of 20.2 months.

Selpercatinib targets both malignant cells and healthy and can produce severe adverse effects such as hepatotoxicity, interstitial lung disease, pneumonitis, hypertension, QT interval prolongation, hemorrhagic events, tumor lysis syndrome, hypothyroidism, and impaired wound healing. Severe and serious adverse events occurred in approximately 60% of patients, and 3% suffered fatal adverse effects.

Producers recommend avoiding the concomitant use of CYP2C8 and CYP3A substrates, as selpercatinib raises the bioavailability of these drugs, increasing the risk of adverse effects. The use of CYP3A inducers and CYP3A inhibitors is also not recommended. 


FDA approves Lilly’s Retevmo® (selpercatinib), the first and only RET inhibitor for adults with advanced or metastatic solid tumors with a RET gene fusion, regardless of type. Press release. Eli Lilly and Company; September 21, 2022.