Takeda’s First-in-Class Oral Therapy Exkivity (Mobocertinib) Receives FDA Accelerated Approval for EGFR Exon20 Insertion-Positive NSCLC

Today, Takeda announced that the U.S. FDA has granted accelerated approval to its oral tyrosine kinase inhibitor Exkivity (mobocertinib) for the treatment of patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring EGFR exon 20 insertion mutations, whose disease has progressed on or after platinum-based chemotherapy. The press release noted that this is the first oral therapy specifically designed and approved by the U.S. FDA for NSCLC patients with EGFR exon 20 insertion mutations.

The FDA approval was based on a patient population that had previously received platinum-based chemotherapy in a Phase 1/2 clinical trial. Data presented at this year’s ASCO Annual Meeting showed that, according to the assessment by an independent data monitoring committee, mobocertinib achieved a confirmed objective response rate of 28%, a median duration of response of 17.5 months, a median overall survival of 24 months, and a median progression-free survival of 7.3 months.

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

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 the World Health Organization. Patients with EGFR exon 20 insertion-positive metastatic NSCLC account for approximately 1–2% of all NSCLC cases, and this condition is more prevalent in Asian populations. These patients have a poorer prognosis compared to those with other EGFR mutations. Existing EGFR tyrosine kinase inhibitors and chemotherapy provide limited clinical 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 FDA for the treatment of patients with EGFR exon 20 insertion-positive metastatic non-small cell lung cancer (NSCLC) who experience 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. In May this year, it was proposed for priority review in China.

References:

[1] Takeda’s EXKIVITY™ (mobocertinib) Approved by U.S. FDA as the First Oral Therapy Specifically Designed for Patients with EGFR Exon20 Insertion+ NSCLC. Retrieved September 15, 2021, from https://www.businesswire.com/news/home/20210915006101/en

[2] 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

(Original text edited)

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