FDA Approves Pralsetinib for RET-Altered Thyroid Cancers
On December 1, 2020, the FDA approved pralsetinib for adult and pediatric patients with advanced or metastatic RET-mutant medullary thyroid cancer requiring systemic therapy or RET-fusion positive thyroid cancer requiring systemic therapy and who are radioactive iodine-refractory. This application was granted accelerated approval based on overall response rate (ORR) and duration of response.
This approval was based on efficacy in a multicenter, open label, multi-cohort clinical trial in patients whose tumors had RET gene alterations.
The overall response rate in 55 patients with metastatic RET-mutant MTC who previously received cabozantinib or vandetanib was 60%, with 79% of responding patients having responses lasting 6 months or longer. Among 29 patients with RET-mutant MTC who did not receive cabozantinib or vandetanib, the ORR was 66% with 84% of these patients having responses last 6 months or longer. The ORR was 89% in 9 patients with RET fusion-positive thyroid cancer who were radioactive iodine-refractory, all responding patients had a response lasting 6 months or longer.
The most common (≥25%) adverse events associated with pralsetinib use were constipation, hypertension, fatigue, musculoskeletal pain, and diarrhea. —Kaitlyn Manasterski
Source: US Food and Drug Administration. FDA approves pralsetinib for RET-altered thyroid cancers. Updated December 01, 2020. https://www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-pralsetinib-ret-altered-thyroid-cancers . Accessed December 02, 2020.