Therapeutic efficacy of Pralsetinib

Based on the preliminary data of objective response rate (ORR) and duration of response (DOR) from 114 enrolled patients in the ARROW trial (Trial No.: NCT03037385), pralsetinib was granted accelerated approval on September 4, 2020, for the treatment of non-small cell lung cancer (NSCLC). This trial is a multicenter, open-label, multi-cohort clinical study. Subsequently, by incorporating data from an additional 123 patients and extending the follow-up period to 25 months to evaluate the durability of the response, the product was ultimately approved for regular marketing.

The efficacy data were derived from a total of 237 patients with locally advanced or metastatic RET fusion-positive non-small cell lung cancer. Patients received continuous pralsetinib treatment until disease progression or the occurrence of intolerable toxic reactions.

The primary efficacy endpoints were objective response rate (ORR) and duration of response (DOR) as assessed by the Blinded Independent Review Committee (BIRC). Among the 107 treatment-naive patients, the objective response rate reached 78% (95% confidence interval [CI]: 68%–85%), with a median duration of response of 13.4 months (95% CI: 9.4–23.1 months); among the 130 patients previously treated with platinum-based chemotherapy, the objective response rate was 63% (95% CI: 54%–71%), with a median duration of response of 38.8 months (95% CI: 14.8 months–not reached).

from FDA，2024.03