Clin Cancer Res 2021 May 27. Epub 2021 May 27.
Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.
The FDA granted accelerated approval for pralsetinib on September 4, 2020 for non-small cell lung cancer (NSCLC) and December 1, 2020 for thyroid cancer, for: (i) adult patients with metastatic fusion-positive NSCLC, (ii) adult and pediatric patients ≥12 years of age with advanced or metastatic -mutant medullary thyroid cancer who require systemic therapy, and (iii) adult and pediatric patients ≥12 years of age with advanced or metastatic fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine refractory (if radioactive iodine is appropriate). Approval was based on the results of a multicenter, open-label, multi-cohort clinical trial (ARROW, NCT03037385), demonstrating substantial overall response rates (ORR) and durable responses in patients with altered tumors. ORRs within the approved patient populations ranged from 57% [95% confidence interval (CI), 46-68] in patients with fusion-positive NSCLC previously treated with platinum chemotherapy to 89% (95% CI, 52-100) in patients with fusion-positive thyroid cancer, with response duration of at least 6 months in most responders. Read More