The Food and Drug Administration (FDA) has granted Priority Review to mobocertinib (TAK-788; Takeda Pharmaceutical) for the treatment of adults with epidermal growth factor receptor (EGFR) Exon20 insertion mutation-positive (insertion+) metastatic non-small cell lung cancer (mNSCLC), as detected by an FDA-approved test, who have received prior platinum-based chemotherapy.

Mobocertinib is an investigational, first-in-class, oral tyrosine kinase inhibitor designed to selectively target EGFR Exon20 insertion mutations. The NDA is supported by data from a multicenter, open-label phase 1/2 trial ( NCT02716116) that evaluated the efficacy and safety of mobocertinib 160mg once daily in 114 adults with EGFR Exon20 insertion+ mNSCLC whose disease progressed on or after platinum-based chemotherapy.

Results demonstrated an objective response rate (ORR) of 35% (n=40; 95% CI, 26-45) as assessed by investigator, and 28% (n=32; 95% CI, 20-37) as assessed by an independent review committee (IRC). Median duration of response, median progression-free survival, and disease control rate were 17.5 months (95% CI, 7.4-20.3), 7.3 months (95% CI, 5.5-9.2), and 78% (n=89; 95% CI, 69-85), respectively, as assessed by IRC.

As for safety, the most common treatment-emergent adverse events (incidence ≥20%) were diarrhea (90%), rash (45%), paronychia (34%), nausea (32%), decreased appetite (32%), dry skin (30%), and vomiting (30%). There were 19 patients who discontinued treatment because of side effects, most commonly due to diarrhea and nausea.

“Patients with EGFR Exon20 insertion+ mNSCLC face considerable challenges, as current treatment options provide limited benefit, resulting in poor survival outcomes,” said Christopher Arendt, head, Oncology Therapeutic Area Unit, Takeda. “We are excited to be one step closer to offering mobocertinib as an effective oral therapy for NSCLC patients with EGFR Exon20 insertions that have received prior platinum-based chemotherapy and look forward to continuing conversations with regulatory agencies in the US and around the globe.”

A Prescription Drug User Fee Act target date of October 26, 2021 has been set for the application.

The Company has also established an Expanded Access Program ( NCT04535557) for patients in the US who may be eligible to receive access to mobocertinib while the NDA is under review.


  1. Takeda announces U.S. FDA grants Priority Review for New Drug Application for mobocertinib (TAK-788) as a treatment for EGFR Exon20 insertion+ metastatic non-small cell lung cancer. [press release]. Cambridge, MA & Osaka, Japan: Takeda Pharmaceutical Company Limited; April 27, 2021. 
  2. Takeda presents positive results for mobocertinib in patients with EGFR Exon20 insertion+ mNSCLC who received prior platinum-based chemotherapy. [press release]. Cambridge, MA & Osaka, Japan: Takeda Pharmaceutical Company Limited; January 28, 2021.