Recently, the U.S. Food and Drug Administration (FDA) granted Priority Review designation to treatments for surgically resected, high-risk, muscle-invasive urothelial carcinoma and epidermal growth factor receptor exon 20 insertion mutation–positive metastatic non–small cell lung cancer (NSCLC).
Priority Review for Nivolumab as Adjuvant Treatment for Patients With Muscle-Invasive Urothelial Carcinoma
The FDA accepted a supplemental biologics license application for nivolumab for the adjuvant treatment of patients with surgically resected, high-risk, muscle-invasive urothelial carcinoma. The FDA granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of September 3, 2021.
The filing was based on results from the pivotal CheckMate 274 trial, a phase III, randomized, double-blind, multicenter study evaluating nivolumab compared to placebo in patients with muscle-invasive urothelial cancer at a high risk of recurrence after radical surgery. A total of 709 patients were randomly assigned 1:1 to receive nivolumab at 240 mg or placebo every 2 weeks for up to 1 year. The primary endpoints of the trial are disease-free survival in all randomly assigned patients (ie, the intention-to-treat population) and in the subset of patients whose tumors express PD-L1 ≥ 1%. Key secondary endpoints include overall survival, non–urothelial tract recurrence-free survival, and disease-specific survival.
In the trial, nivolumab demonstrated a statistically significant and clinically meaningful increase in disease-free survival vs placebo, regardless of patients’ PD-L1 expression levels. Nivolumab was generally well tolerated, with a safety profile that was consistent with previously reported studies in patients with solid tumors. Results from the primary analysis of CheckMate 274 were presented in an oral presentation during the Genitourinary Cancers Symposium in February 2021 (Abstract 391).
Priority Review for Mobocertinib in EGFR Exon 20 Insertion–Positive Metastatic NSCLC
The FDA granted Priority Review for a new drug application for mobocertinib in the treatment of adult patients with epidermal growth factor receptor (EGFR) exon 20 insertion mutation–positive metastatic NSCLC, as detected by an FDA-approved test, who have received prior platinum-based chemotherapy. The application was submitted under the FDA’s accelerated approval program. The review is being conducted under Project Orbis, an initiative of the FDA Oncology Center of Excellence, which provides a framework for concurrent submission and review of oncology products among international partners.
Mobocertinib is an investigational, first-in-class, oral tyrosine kinase inhibitor specifically designed to selectively target EGFR exon 20 insertion mutations. The application for mobocertinib is primarily based on results from a phase I/II trial, which is evaluating the safety and efficacy of oral mobocertinib in patients with metastatic NSCLC. The trial comprises a phase I dose escalation, evaluation of mobocertinib as a monotherapy and in combination with chemotherapy, several expansion cohorts, and an extension cohort in patients with EGFR exon 20 insertion–positive metastatic NSCLC. The platinum-pretreated population efficacy analysis investigated 114 patients who received prior platinum-based therapy in the phase I/II trial and were treated with mobocertinib at the 160-mg once-daily dose.
Takeda has established an Expanded Access Program (NCT04535557) for patients in the United States who may be eligible to receive access to mobocertinib during the review of the new drug application.
