FDA Grants Regular Approval to Pralsetinib in Advanced NSCLC

The approval is based on results from the phase 1/2 ARROW trial (NCT03037385), which assessed the use of pralsetinib in patients with thyroid cancer, NSCLC, and other advanced solid tumors.

The FDA has granted regular approval to pralsetinib (Gavreto) for the treatment of metastatic RET fusion-positive non–small cell lung cancer (NSCLC) detected with an approved test, according to a press release from the organization.¹

The approval is based on results from the phase 1/2 ARROW trial (NCT03037385), which assessed the use of pralsetinib in patients with thyroid cancer, NSCLC, and other advanced solid tumors.² In September 2020, the FDA granted accelerated approval to pralsetinib based on results from 114 patients.³ With results from an additional 123 patients and 25 months of follow-up, regular approval was granted.

A total of 237 received treatment until disease progression or unacceptable toxicity. Per Blinded Independent Review Committee assessment, the overall response rate (ORR) was 78% (95% CI, 68%-85%), and the median duration of response (DOR) was 13.4 months (95% CI, 9.4-23.1). Patients who were previously given platinum-based chemotherapy (n = 130) had an ORR of 63% (95% CI, 54%-71%) and a median DOR of 38.8 months (95% CI, 14.8-not estimable).

Of note, the most common adverse effects included musculoskeletal pain, constipation, hypertension, diarrhea, fatigue, edema, pyrexia, and cough.

For patients to be enrolled on the study, they must have had pathologically and definitively diagnosed non-resectable advanced solid tumor, oncogenic RET-rearrangement/fusion or mutation, agreed to provide tumor tissue, and had an ECOG status of 0 or 1.

Patients were not eligible for enrollment if their cancer had a driver mutation other than RET, significant or uncontrolled cardiovascular disease, or a major surgical procedure within 14 days of the first dose.

References

1. FDA approves pralsetinib for non-small cell lung cancer with RET gene fusions. News release. FDA. August 9, 2023. Accessed August 9, 2023. https://bit.ly/47r5hYd
2. Subbiah V, Cassier PA, Siena S, et al. Pan-cancer efficacy of pralsetinib in patients with RET fusion-positive solid tumors from the phase 1/2 ARROW trial. Nat Med. Published Online August 12, 2022. doi:10.1038/s41591-022-01931-y
3. Blueprint Medicines announces FDA approval of GAVRETO™ (pralsetinib) for the treatment of adults with metastatic RET fusion-positive non-small cell lung cancer. News release. Blueprint Medicines. September 5, 2020. Accessed August 9, 2023. https://bit.ly/3DPT3uF