FDA Approves Mobocertinib for NSCLC with EGFR Exon 20 Insertion Mutations


The FDA has green lit mobocertinib for the treatment of adult patients with locally advanced or metastatic non-small cell lung cancer with an EGFR exon 20 insertion mutation.

The FDA has approved mobocertinib (Exkivity) for the indication of adult patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) whose disease harbors an epidermal growth factor receptor (EGFR) exon 20 insertion mutation as detected by an FDA-approved test, either following or beyond platinum-based chemotherapy.

This first-in-class, oral tyrosine kinase inhibitor (TKI) is the first approved oral therapy engineered to target EGFR exon 20 insertion mutations, according to Takeda Pharmaceutical Company Limited, the manufacturer of the small-molecule TKI.1

“EGFR Exon20 insertion+ NSCLC is an underserved cancer that we have been unable to target effectively with traditional EGFR TKIs,” said Pasi A. Jänne, MD, PhD, Dana Farber Cancer Institute, in a press release. “The approval of EXKIVITY (mobocertinib) marks another important step forward that provides physicians and their patients with a new targeted oral therapy specifically designed for this patient population that has shown clinically meaningful and sustained responses.”

The regulatory decision is based on findings from the phase 1/2 trial of Exkivity. In the trial, 114 patients with EGFR Exon20 insertions who had been previously treated with platinum-based therapy received mobocertinib at a 160-mg dosage. Findings revealed an overall response rate (ORR) of 28% through an independent review committee (IRC) and 35% through investigator analysis. The median duration of response (DOR) was 17.5 months. The median overall survival (OS) was 24 months, and the median progression-free survival (PFS) was 7.3 months. DOR, OS, and PFS were all determined by IRC.

Notable adverse events from the study drug included diarrhea, rash, nausea, stomatitis, vomiting, decreased appetite, paronychia, fatigue, dry skin, and musculoskeletal pain. Patients and providers should be aware that there is a risk of interstitial lung disease/pneumonitis, cardiac toxicity, and diarrhea associated with the drug.

“Patients with EGFR Exon20 insertion+ NSCLC have historically faced a unique set of challenges living with a very rare lung cancer that is not only underdiagnosed, but also lacking targeted treatment options that can improve response rates,” added Marcia Horn, executive director, Exon 20 Group at ICAN, International Cancer Advocacy Network. “As a patient advocate working with EGFR Exon20 insertion+ NSCLC patients and their families every day for nearly five years, I am thrilled to witness continued progress in the fight against this devastating disease and am grateful for the patients, families, healthcare professionals and scientists across the globe who contributed to the approval of this promising targeted therapy.”

A confirmatory trial will determine continued approval for the indication.


Takeda’s Exkivity (mobocertinib) approved by US FDA as the first oral therapy specifically designed for patients with EGFR Exon20 insertion _NSCLC. Takeda. News release. Accessed September 15, 2021. https://www.businesswire.com/news/home/20210915006101/en/Takeda%E2%80%99s-EXKIVITY%E2%84%A2-mobocertinib-Approved-by-U.S.-FDA-as-the-First-Oral-Therapy-Specifically-Designed-for-Patients-with-EGFR-Exon20-Insertion-NSCLC

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