Takeda's First-in-Class Therapy Mobocertinib Receives FDA Priority Review for EGFR Exon20 Insertion-Positive Metastatic Non-Small Cell Lung Cancer

Takeda Announces FDA Priority Review for Investigational Therapy Mobocertinib (TAK-788) for Adult Patients with Metastatic Non-Small Cell Lung Cancer (NSCLC) Harboring EGFR Exon 20 Insertions Who Have Previously Received Platinum-Based ChemotherapyToday, Takeda announced that the U.S. Food and Drug Administration (FDA) has granted priority review to its new drug application for the investigational therapy mobocertinib (TAK-788) for the treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) positive for EGFR exon 20 insertions who have previously received platinum-based chemotherapy. The press release noted that mobocertinib is the first oral therapy specifically designed to selectively target EGFR exon 20 insertions.

Non-small cell lung cancer (NSCLC) is the most common type of lung cancer, accounting for approximately 85% of newly diagnosed lung cancer cases globally each year, according to World Health Organization statistics. Patients with metastatic NSCLC harboring EGFR exon 20 insertion mutations account for approximately 1–2% of all NSCLC patients, and this condition is more prevalent in Asian populations. These patients have a poorer prognosis compared to those with other EGFR mutations. Currently, there are no approved targeted therapies for this genetic alteration, and existing EGFR tyrosine kinase inhibitors and chemotherapy offer limited benefit to these patients.

Mobocertinib is a potent oral small-molecule tyrosine kinase inhibitor specifically designed to selectively target EGFR exon 20 insertion mutations. In April 2020, it was granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) positive for EGFR exon 20 insertions who have experienced disease progression during or after platinum-based chemotherapy. In October 2020, mobocertinib was designated as a Breakthrough Therapy Product by the Center for Drug Evaluation (CDE) in China.

The granting of priority review eligibility was based on the results of the Phase 1/2 clinical trial. Previously announced results showed that, among 114 patients, mobocertinib achieved a confirmed objective response rate (ORR) of 28%, with a median duration of response of 17.5 months and a disease control rate (DCR) of 78%, as assessed by an independent data monitoring committee.

▲Phase 1/2 Clinical Trial Results of Mobocertinib (Image source: Reference [2])

References:

[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. Retrieved April 27, 2021, from https://www.businesswire.com/news/home/20210427006162/en

[w] Takeda Presents Positive Results For Mobocertinib in Patients with EGFR Exon20 insertion+ mNSCLC Who Received Prior Platinum-based Chemotherapy. Retrieved January 28, 2021, from https://www.businesswire.com/news/home/20210128005072/en

Note: This article aims to introduce advances in medical and health research and does not constitute treatment recommendations. For guidance on treatment plans, please consult a qualified healthcare provider at a reputable hospital.

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