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 assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.
Posted: August 2023
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