The following is a message from the Director of the FDA Oncology Center of Excellence, Dr. Richard Pazdur:
On August 9, 2023, the Food and Drug Administration granted regular approval to pralsetinib (Gavreto, Genentech, Inc.) for adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test.
Pralsetinib was previously granted accelerated approval for the NSCLC indication on Sept. 4, 2020, based on initial overall response rate (ORR) and duration of response (DOR) in 114 patients enrolled in the ARROW trial (NCT03037385), a multicenter, open-label, multi-cohort trial. The conversion to regular approval was based on data from an additional 123 patients and 25 months of additional follow-up to assess durability of response.
Efficacy was demonstrated in a total of 237 patients with locally advanced or metastatic RET fusion-positive NSCLC. Patients received pralsetinib until disease progression or unacceptable toxicity.
The primary efficacy measures were ORR and DOR as determined by a Blinded Independent Review Committee (BIRC). Among 107 treatment-naïve patients, ORR was 78% (95% CI: 68, 85) with a median DOR of 13.4 months (95% CI: 9.4, 23.1). Among 130 patients previously treated with platinum-based chemotherapy, ORR was 63% (95% CI: 54, 71) with a median DOR of 38.8 months (95% CI: 14.8, not estimable).
The most common adverse reactions (≥ 25%) were musculoskeletal pain, constipation, hypertension, diarrhea, fatigue, edema, pyrexia, and cough.
The recommended pralsetinib dose is 400 mg orally once daily. Pralsetinib is taken on an empty stomach (no food intake for at least 2 hours before and at least 1 hour after taking pralsetinib).
This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.
This application was granted breakthrough designation and orphan drug designation. FDA expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.
For information on the COVID-19 pandemic, see the following resources:
- FDA: Coronavirus Disease 2019 (COVID-19)
- NCI: Coronavirus: What People With Cancer Should Know
- CDC: Coronavirus (COVID-19)
Please visit the FDA Hematology/Oncology (Cancer) Approvals & Safety Notifications webpage for a list of current and past approvals.