On December 1, 2020, the FDA granted approval to pralsetinib (Gavreto, Blueprint Medicines Corporation) for patients with advanced or metastatic RET-mutant medullary thyroid cancer or RET fusion-positive thyroid cancer requiring systemic therapy.
The efficacy of pralsetinib was demonstrated in patients with RET alterations enrolled on the ARROW trial. The main efficacy outcome measures were overall response rate (ORR) and response duration per blinded independent review committee using RECIST 1.1.
For advanced or metastatic RET-mutant medullary thyroid cancer, efficacy was evaluated in 55 patients previously treated with cabozantinib or vandetanib. Among these patients, the ORR was 60% with 79% of responses lasting ≥6 months. Additionally, efficacy was evaluated in 29 patients with RET-mutant medullary thyroid cancer who did not receive prior cabozantinib or vandetanib. Among these patients, the ORR was 66% with 84% of responses lasting ≥6 months.
For RET fusion-positive thyroid cancer, efficacy was evaluated in 9 patients who were radioactive iodine-refractory. The ORR was 89% with all responses lasting ≥6 months.
The most common adverse evets (≥25%) were constipation, hypertension, fatigue, musculoskeletal pain, and diarrhea.—Janelle Bradley
Source: US Food and Drug Administration. FDA approves pralsetinib for RET-altered thyroid cancers. December 1, 2020. https://www.fda.gov/drugs/drug-approvals-and-databases/fda-approves-pralsetinib-ret-altered-thyroid-cancers. Accessed December 1, 2020
