Previously, the FDA had granted breakthrough therapy and orphan drug designations for selpercatinib to treat certain patients with advanced RET fusion–positive thyroid cancer and those with RET-altered non–small-cell lung cancer (NSCLC) or medullary thyroid cancer (MTC). The agency also granted orphan drug designation for the treatment of patients with RET fusion–positive NSCLC, RET-mutant MTC, and RET fusion–positive thyroid cancer.
“We are pleased the FDA granted priority review status for the NDA for selpercatinib. This represents an important step toward providing a new precision therapy for people living with certain RET-driven cancers,” stated Anne E. White, President, Lilly Oncology, in a company press release.
Key Insights:
- The New Drug Application (NDA) for selpercatinib includes data from the phase 1/2 LIBRETTO-001 clinical trial, the largest study of patients with RET-driven lung and thyroid cancers treated with an RET inhibitor. Data from phase 2 of the study showed an overall response rate (ORR) of 77% in evaluable patients with RET fusion–positive cancers. In 2019, updated data showed an ORR of 68% in patients with lung cancer and an ORR of 56% in patients with thyroid cancer.
- Approximately 2% of patients with NSCLC, 10% to 20% of patients with papillary and other thyroid cancers, and patients with a subset of other cancers have RET fusions. Activating RET point mutations account for approximately 60% of MTC. RET fusion cancers and RET-mutant MTC depend on this single activated kinase for their uncontrolled growth.
- In December 2019, Lilly Oncology opened 2 phase 3 trials: LIBRETTO-431, which will evaluate selpercatinib in treatment-naïve patients with RET fusion–positive NSCLC, and LIBRETTO-531, which will evaluate selpercatinib in treatment-naïve patients with RET-mutant MTC. Each trial will enroll 400 patients.
