September 2021: The FDA granted accelerated approval to mobocertinib (Exkivity, Takeda Pharmaceuticals, Inc.) for adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) who have EGFR exon 20 insertion mutations, as detected by an FDA-approved test, and whose disease has progressed on or after platinum-based chemotherapy.
The Oncomine Dx Target Test (Life Technologies Corporation) was also approved by the FDA today as a companion diagnostic device for selecting patients with the aforesaid mutations for mobocertinib treatment.
Study 101, a non-randomized, open-label, multicohort clinical trial (NCT02716116) that included patients with locally advanced or metastatic NSCLC who had EGFR exon 20 insertion mutations, received FDA approval. The efficacy of 114 patients whose illness had worsened during or after platinum-based treatment was assessed. Patients were given mobocertinib 160 mg orally once a day until the disease progressed or effects became unbearable.
Overall response rate (ORR) according to RECIST 1.1 as assessed by blinded independent central review (BICR) and response duration were the key efficacy outcome measures. With a median response time of 17.5 months, the ORR was 28 percent (95 percent CI: 20 percent, 37 percent) (95 percent CI: 7.4, 20.3).
Diarrhea, rash, nausea, stomatitis, vomiting, decreased appetite, paronychia, weariness, dry skin, and musculoskeletal pain were the most prevalent side effects (>20 percent). A boxed warning for QTc prolongation and Torsades de Pointes is included on the product label, as well as cautions for interstitial lung disease/pneumonitis, cardiac toxicity, and diarrhoea.
Until disease progression or severe toxicity, the recommended mobocertinib dose is 160 mg orally once daily.