FDA Approves First Oral Treatment for EGFR Exon 20+ NSCLC

The FDA approved Exkivity to treat non-small cell lung cancer along with Thermo Fisher Scientific’s Oncomine DX Target Test, which is intended to be a companion diagnostic for the treatment.

The FDA has announced the approval of mobocertinib (Exkivity) for adults with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy. The FDA also simultaneously approved Thermo Fisher Scientific’s Oncomine Dx Target Test as a companion diagnostic for mobocertinib to identify patients with NSCLC harboring EGFR exon 20 insertions.

Mobocertinib is the first and only approved oral therapy drug specifically designed for EGFR exon 20 insertion mutations.

“The approval of Exkivity introduces a new and effective treatment option for patients with EGFR Exon 20 insertion+ NSCLC, fulfilling an urgent need for this difficult-to-treat cancer,” Teresa Bitetti, president of the global oncology business unit at Takeda, said in a press release. “Exkivity is the first and only oral therapy specifically designed to target EGFR Exon 20 insertions, and we are particularly encouraged by the duration of the responses observed with a median of approximately 1.5 years. This approval milestone reinforces our commitment to meeting the needs of underserved patient populations within the oncology community.”

The approval was based on results from a phase 1/2 trial of 114 patients with EGFR exon 20 insertion+ NSCLC who received prior platinum-based therapy and were treated with mobocertinib at a dose of 160 mg.

The trail showed showed that mobocertinib generated a confirmed overall response rate of 28% (95% CI, 20%-37%) per independent review committee assessment, with a median duration of response of 17.5 months (95% CI, 7.4-20.3). Further, median progression-free survival achieved with mobocertinib in this patient group was 7.3 months (95% CI, 5.5-9.2), and the median overall survival was 24.0 months (95% CI, 14.6-28.8).

Common adverse events include diarrhea, rash, nausea, vomiting, fatigue. Warnings and precautions include lung disease, pneumonitis, cardiac toxicity, and diarrhea.

Mobocertinib was previously granted priority review and received Breakthrough Therapy Designation, Fast Track Designation, and Orphan Drug Designation from the FDA.

Continued approval for this indication may be contingent upon verification and description of a clinically beneficial confirmatory trial.

“EGFR Exon20 insertion+ NSCLC is an underserved cancer that we have been unable to target effectively with traditional EGFR TKIs,” Pasi Jänne, MD, PhD, from the Dana Farber Cancer Institute, said in the press release. “The approval of Exkivity 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.”


Takeda’s EXKIVITY™ (mobocertinib) approved by U.S. FDA as the first oral therapy specifically designed for patients with EGFR Exon20 Insertion+ NSCLC. Business Wire. News release. September 15, 2021. Accessed September 16, 2021. https://www.businesswire.com/news/home/20210915006101/en