The U.S. Food and Drug Administration (FDA) has approved Gavreto (pralsetinib) for the treatment of patients with advanced or metastatic rearranged during transfection (RET)-mutant medullary thyroid cancer (MTC) who require systemic therapy, or with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate), according to a press release from Roche.
The FDA’s approval applies to adult and pediatric patients 12 years of age and older. Roche said these indications were approved under the FDA’s accelerated approval program based on data from the phase I/II ARROW study. Continued approval for these indications may be contingent upon verification and description of clinical benefit in confirmatory trials.
Approximately 10-20 percent of people with papillary thyroid cancer (the most common type of thyroid cancer)1 have RET fusion-positive tumours,2 and roughly 90 percent of people with advanced MTC (a rare form of thyroid cancer) carry RET mutations. Biomarker testing for RET fusions and mutations can help identify people who are eligible for treatment with Gavreto.
Roche said the approvals are based on results from the phase I/II ARROW study, which demonstrated durable clinical activity in people with or without prior therapy and regardless of RET alteration genotypes. Treatment with Gavreto led to an overall response rate (ORR) of 60 percent in 55 people with RET-mutant metastatic MTC previously treated with cabozantinib and/or vandetanib, and the median duration of response (DoR) was not reached.
In 29 people with cabozantinib- and vandetanib-naive RET-mutant advanced MTC who were determined to be not eligible for standard therapies, the ORR was 66 percent, and the median DoR was not reached. In nine people with RET fusion-positive metastatic thyroid cancer, Gavreto demonstrated an ORR of 89 percent, and the median DoR was not reached.
