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FDA Grants Priority Review to Mobocertinib for Advanced EGFR Exon 20–Mutant NSCLC

*April 2021*

The FDA has granted a priority review designation to a new drug application (NDA) for mobocertinib (TAK-788) for the treatment of adult patients with EGFR exon 20 insertion mutation–positive metastatic non–small cell lung cancer (NSCLC), as detected via an FDA-approved assay, who have received previous platinum-based chemotherapy.1

The designation is based primarily on findings from a phase 1/2 trial, in which mobocertinib elicited a confirmed objective response rate (ORR) of 26% (95% CI, 19%-35%) per an independent review committee (IRC) and a 35% investigator-assessed ORR (95% CI, 26%-45%).2 Additionally, the median progression-free survival (PFS) was 7.3 months and was from both IRC and investigator assessment.

Mobocertinib marks the first oral treatment that can selectively target the patient population of NSCLC with EGFR exon 20 insertion mutations. Under the Prescription Drug User Fee Act, the FDA will make a decision on the NDA by October 26, 2021.

“Patients with EGFR exon 20 insertion–positive [metastatic] NSCLC face considerable challenges, as current treatment options provide limited benefit, resulting in poor survival outcomes,” said Christopher Arendt, head, Oncology Therapeutic Area Unit, of Takeda, the developer of mobocertinib. “We are excited to be one step closer to offering mobocertinib as an effective oral therapy for NSCLC patients with EGFR exon 20 insertions that have received prior platinum-based chemotherapy and look forward to continuing conversations with regulatory agencies in the U.S. and around the globe.”

Mobocertinib, an investigational, first-in-class, oral TKI, was previously granted breakthrough therapy designation by the FDA in April 2020 as a potential treatment for this patient population. Additionally, Takeda established an Expanded Access Program for patients in the United States who could receive mobocertinib during the FDA’s review of the application. Read more.