FDA Approves Selpercatinib for RET Fusion–Positive Locally Advanced or Metastatic Solid Tumors

Patients with RET fusion–positive locally advanced or metastatic solid tumors can now receive treatment with selpercatinib following its approval by the FDA.

Selpercatinib has been granted approval by the FDA at an oral dose of 40 mg and 80 mg for patients with RET fusion–positive locally advanced or metastatic solid tumors following progression on or after previous systemic therapy with no other adequate treatment options, according to a press release from the FDA.

The continued approval of selpercatinib in this indication may depend on confirmation of clinical benefit from a trial. The phase 1/2 LIBRETTO-001 trial (NCT03157128) included a total of 41 patients with solid malignancies, with the most common tumor types being pancreatic adenocarcinoma (27%), colorectal cancer (24%), salivary gland cancer (10%), and unknown primary disease (7%). The overall response rates in each of these tumor types were 55%, 20%, 50%, and 33%, respectively. Additionally, the durations of response in each respective group were 2.5, 38.3+ months; 5.6, 13.3 months; 5.7, 28.8+ months; and 9.2 months.

“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," co-investigator for LIBRETTO-001 Vivek Subbiah, MD, associate professor of Investigational Cancer Therapeutics at the University of Texas MD Anderson Cancer Center, said in the press release. “These data and FDA approval of the tumor-agnostic indication underscore the importance of routine, comprehensive genomic testing for patients across a wide variety of tumor types.”


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. News release. Eli Lilly and Company. September 21, 2022. Accessed September 22, 2022. https://prn.to/3dFC8Bz