Update: On September 21, 2022, the U.S. Food and Drug Administration (FDA) expanded the approval of selpercatinib (RetevmoTM) beyond treating lung and thyroid cancers. The drug is now approved for adults with any locally advanced or metastatic solid tumor carrying a RET gene fusion that has not responded to other treatments. The FDA approval was based on results from the LIBRETTO-001 trial, a multicenter study overseen by medical oncologist Alexander Drilon, Chief of the Memorial Sloan Kettering Cancer Center (MSK) Early Drug Development Service.
The U.S. Food and Drug Administration (FDA) recently granted accelerated approval to selpercatinib (Retevmo™) for lung and thyroid cancers with RET (REarranged during Transfection) gene mutations or fusions. Memorial Sloan Kettering’s Alexander Drilon, MD, Acting Chief, Early Drug Development Service, served as principle investigator of the LIBRETTO-001 Phase 1/2 trial of the of the targeted therapy drug, which formed the basis of the FDA new drug application submission. MSK’s Eric Sherman, MD, medical oncologist, served as co-principle investigator. This was the largest trial ever reported in patients with RET-driven cancers. Approval was based on the trial’s endpoints of objective response rate (ORR) and duration of response (DoR).
“The approval of selpercatinib marks another important step in the field of precision oncology and continues to support the need for more advanced genomic profiling,” explained Dr. Drilon. “RET-driven cancers are now specifically targetable in the same manner as activating EGFR and ALK alterations, across all lines of therapy. This approval offers hope to a patient population who previously had no targeted treatment options available.”
Selpercatinib (previously known as LOXO-292) is an oral and highly selective drug developed for the treatment of patients with cancers that harbor abnormalities in the RET gene. Genomic alterations involving RET, which include fusions and activating point mutations, lead to overactive RET signaling and uncontrolled cell growth. There were previously no targeted therapies approved for this patient population. Selpercatinib was granted Breakthrough Therapy Designation by the FDA in 2018 based on initial data from the LIBRETTO-001 trial. Data from this trial was featured at the 2019 World Congress on Lung Cancer and the 2019 European Society for Medical Oncology annual meeting. Selpercatinib follows other landmark targeted therapy approvals.