FDA Approves Pralsetinib for RET-Mutant Medullary Thyroid Cancer

The FDA has approved pralsetinib (Gavreto) for the treatment of adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy or RET fusion-positive thyroid cancer who require systemic therapy and who are refractory to radioactive iodine.¹

The regulatory decision was based on data from the multicenter, open label, multicohort phase 1/2 ARROW trial (NCT03037385), where the safety and efficacy of pralsetinib was evaluated in patients with RET fusion—positive non–small cell lung cancer (NSCLC), RET-mutated MTC, RET fusion–positive thyroid cancer, and other RET-altered solid tumors.

Investigators prospectively identified these alterations in local laboratories through the use of next-generation sequencing, fluorescence in situ hybridization, or other tests. The main efficacy outcome measures evaluated in the trial consisted of overall response rate (ORR) and duration of response (DOR) per a blinded independent review committee via RECIST v1.1 criteria. Results showed that among 55 patients with advanced or metastatic RET-mutant MTC who had received prior cabozantinib (Cabometyx) or vandetanib, pralsetinib elicited an ORR of 60% (95% CI, 46%-73%). Additionally, 79% percent of responders experienced responses that persisted for 6 months or longer.

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