BRIEF—Takeda gains FDA Breakthrough status for mobocertinib

Japan’s Takeda Pharmaceutical today announced that the US Food and Drug Administration has granted Breakthrough Therapy designation for its investigational drug mobocertinib (formerly known as TAK-788).

The drug is intended for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations whose disease has progressed on or after platinum-based chemotherapy.

There are currently no approved therapies designed to treat this specific form of NSCLC.

Mobocertinib is a small-molecule tyrosine kinase inhibitor (TKI) designed to selectively target EGFR and human EGFR 2 (HER2) exon 20 insertion mutations.

The Breakthrough Therapy designation is based on results from a Phase I/II study, which showed mobocertinib yielded a median progression-free survival (PFS) of 7.3 months and a confirmed overall response rate (ORR) of 43% in patients with locally-advanced or metastatic EGFR exon 20 insertion-mutant NSCLC who had been previously treated with systemic chemotherapy.