1. FDA Approval Summary: Selpercatinib for the Treatment of Lung and Thyroid Cancers with RET Gene Mutations or Fusions
- Author
-
Julia A. Beaver, Whitney S. Helms, Harpreet Singh, Jiang Liu, Reena Philip, Diana Bradford, Dun Liang, Francisca Reyes Turcu, Amy M. Skinner, Rosane Charlab, Shenghui Tang, Soma Ghosh, Lauren S. L. Price, Marc R. Theoret, Richard Pazdur, Erin Larkins, Donna Roscoe, Yangbing Li, Jeanne Fourie Zirkelbach, Sirisha Mushti, Autumn Zack-Taylor, Lisa Rodriguez, and Paul G. Kluetz
- Subjects
0301 basic medicine ,Oncology ,Cancer Research ,medicine.medical_specialty ,Lung ,endocrine system diseases ,business.industry ,Thyroid ,Medullary thyroid cancer ,medicine.disease ,QT interval ,Systemic therapy ,Clinical trial ,03 medical and health sciences ,030104 developmental biology ,0302 clinical medicine ,medicine.anatomical_structure ,030220 oncology & carcinogenesis ,Internal medicine ,Toxicity ,medicine ,business ,Thyroid cancer - Abstract
On May 8, 2020, the FDA granted accelerated approval to selpercatinib for (i) adult patients with metastatic RET fusion–positive non–small cell lung cancer (NSCLC), (ii) adult and pediatric patients ≥12 years of age with advanced or metastatic RET-mutant medullary thyroid cancer who require systemic therapy, and (iii) adult and pediatric patients ≥12 years of age with advanced or metastatic RET fusion–positive thyroid cancer who require systemic therapy and who are radioactive iodine refractory (if radioactive iodine is appropriate). Approval was granted on the basis of the clinically important effects on the overall response rate (ORR) with prolonged duration of responses observed in a multicenter, open-label, multicohort clinical trial (LIBRETTO-001, NCT03157128) in patients whose tumors had RET alterations. ORRs within the approved patient populations ranged from 64% [95% confidence interval (CI), 54–73] in prior platinum-treated RET fusion–positive NSCLC to 100% (95% CI, 63–100) in systemic therapy–naïve RET fusion–positive thyroid cancer, with the majority of responders across indications demonstrating responses of at least 6 months. The product label includes warnings and precautions for hepatotoxicity, hypertension, QT interval prolongation, hemorrhagic events, hypersensitivity, risk of impaired wound healing, and embryo-fetal toxicity. This is the first approval of a drug specifically for patients with RET alterations globally.
- Published
- 2020
- Full Text
- View/download PDF