The FDA granted the selective oral tyrosine kinase inhibitor mobocertinib priority review for patients with pretreated metastatic non–small cell lung cancer harboring insertion mutations in EGFR exon 20.



It is for the treatment of patients with metastatic non–small cell lung cancer (mNSCLC) and an EGFR exon 20 insertion mutation who have received prior platinum-based chemotherapy.



EGFR exon 20 insertion[–positive] mNSCLC with current treatment options results in poor survival outcomes.



Studies included 114 patients who received prior platinum-based therapy and were treated as part of the dose-escalation and dose-expansion portions of the phase 1/2 trial. 



Patients were treated with mobocertinib at 160 mg once daily.



The  confirmed objective response rate (ORR) was 26% and 35% by investigator assessment. 



The median progression-free survival (PFS) was 7.3 months, and the median duration of response by IRC was 17.5 months.



Mobocertinib demonstrated clinically meaningful responses and a noteworthy duration of response in patients with EGFR exon 20 insertion–positive metastatic NSCLC who received prior platinum-based therapy.



Treatment-related adverse events (TRAEs) occurring in at least 20% of patients previously treated with platinum chemotherapy included diarrhea (90%), rash (45%), paronychia (34%), nausea (32%), decreased appetite (32%), dry skin (30%), and vomiting (30%). 



Discontinuations due to AEs occurred in 17% of patients, most commonly from diarrhea (4%) and nausea (4%).



Responses in patients who were previously treated with TKIs were also noted.