Analysis Group’s Real-World Data Study with Takeda Supports FDA’s Recent Exkivity^TM (Mobocertinib) Approval

October 19, 2021

An Analysis Group team’s collaborative real-world data (RWD) study contributed to Takeda’s successful bid for the accelerated approval of Exkivity (mobocertinib) by the US Food and Drug Administration (FDA) for the treatment of locally advanced or metastatic non-small cell lung cancer with epidermal growth factor receptor (EGFR) exon 20 insertion mutations. Exkivity is the first and only approved oral targeted therapy specifically designed for this difficult-to-treat cancer.

A team including Managing Principal Mei Sheng Duh, M.P.H., Sc.D., Vice President Maral DerSarkissian, Ph.D., and researchers from Takeda initiated the retrospective RWD analysis of a community-based oncology data source. The analysis complemented Takeda’s Phase 1/2 single-arm, international, open-label, multicohort clinical trial (NCT02716116), also known as Study 101. While the FDA has issued guidance documents for the use of real-world evidence to support new drug applications and biologics license applications, the study team’s expertise on the FDA’s regulatory standard for real-world data was essential in designing a fit-for-purpose study that became a key driver for the successful Exkivity approval.