Full Approval for Targeted Therapy for Lung Cancer  

The FDA granted full approval to pralsetinib as a treatment for certain patients with non-small cell lung cancer  

The U.S. Food and Drug Administration has granted full approval to pralsetinib (Gavreto) to treat adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors carry a genetic alteration known as a RET fusion, in which the RET gene is fused to another gene, as detected by an FDA-approved test. 

The abnormal proteins produced as a result of RET fusion help cancer cells grow. 

Pralsetinib is a molecularly targeted therapy designed to inhibit the function of RET fusion proteins, as well as other mutated forms of RET.  

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Pralsetinib was granted accelerated approval for RET fusion-positive NSCLC in 2020, based on data from ARROW, a multicenter, open-label, multi-cohort phase I/II clinical trial evaluating the efficacy of pralsetinib in patients with NSCLC and other solid tumor types that harbor RET alterations.  

The full approval was based on updated results from the ARROW trial, including data from 123 additional patients and 25 additional months of follow-up to assess durability of response. Efficacy was evaluated in a total of 237 patients with locally advanced or metastatic RET fusion-positive NSCLC who received pralsetinib until disease progression or unacceptable toxicity. 

Overall response rate was 78% among 107 treatment-naïve patients and 63% among 130 patients who had been previously treated with platinum-based chemotherapy. Median duration of response in these two groups was 13.4 months and 38.8 months, respectively. 

NSCLC is the most prevalent type of lung cancer. RET fusions are estimated to be present in 1-2% of NSCLC cases. According to federal statistics, it was estimated that 238,340 new lung cancer cases would be diagnosed, and 127,070 individuals would die of the disease in the U.S. in 2023. 

The FDA rendered its decision on August 9, 2023.