The FDA has approved pralestinib, a once-daily oral therapy, to treat adults with metastatic RET fusion-positive NSCLC, according to a press release from the manufacturer.  

The release noted that the approval was based on data from the Phase I/II Arrow study, which showed an overall response rate of 57% and a complete response rate of 5.7% in a patient population of 87. 

The patients had been previously treated with platinum-based chemotherapy. The median duration of response was not reached.  

The trial also included 27 treatment naïve NSCLC patients with an overall response rate of 70%, and an 11% complete response rate.  

Common adverse reactions included fatigue, constipation, musculoskeletal pain, and increased blood pressure.  

“The FDA approval of Gavreto for RET fusion-positive non-small cell lung cancer is an important step towards our goal of providing an effective treatment option for every person diagnosed with lung cancer, no matter how rare or hard-to-treat their type of disease,” said Levi Garraway, MD, PhD, chief medical officer and head of Global Product Development. 

The FDA also granted Priority Review to pralestinib for the treatment of patients with advanced or metastatic RET-mutant MTC and RET fusion-positive thyroid cancer. A decision on the application is expected by February of next year.  

Approval for pralestinib came under the FDA’s accelerated approval program, and the release noted that continued approval may be contingent on a future confirmatory trial.  


By Adam Hochron