Takeda Pharmaceuticals (Takeda) recently announced that the US Food and Drug Administration (FDA) has granted Mobocertinib (TAK-788) Breakthrough Drug Qualification (BTD) for treatment Patients with metastatic non-small cell lung cancer (NSCLC) who have progressed during or after receiving platinum-containing chemotherapy and carry epidermal growth factor receptor (EGFR) exon 20 insertion mutation. At present, there is no approved treatment plan for this particular type 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 Drug Qualification (BTD) is a new drug review channel created by the FDA in 2012 to speed up the development and review of treatments for serious or life-threatening diseases, and there is preliminary clinical evidence that the drug is comparable to existing therapeutic drugs New drugs that can substantially improve the condition. Obtaining BTD drugs can get more close guidance including senior FDA officials during research and development. It is eligible for rolling review and priority review in new drug market review to ensure that patients are provided with new treatment options in the shortest time.
The FDA awarded mobocertinib BTD based on the overall response rate (ORR) and long-term survival benefit of patients who responded to mobocertinib therapy in a phase I / II study. The study is evaluating the efficacy and safety of mobocertinib (160 mg, once daily orally) in the treatment of patients with locally advanced or metastatic NSCLC who have EGFR exon 20 insertion mutations and have previously received systemic chemotherapy. There are currently no targeted therapies, and the benefits offered by current treatment regimens are limited.
The data from this study marks potentially significant progress in addressing the needs of such patients. The data showed that the median progression-free survival (PFS) of mobocertinib treatment was 7.3 months, and the overall response rate (ORR) was 43% (n = 12/28). In the study, the safety of mobocertinib was controllable (n = 72). The most common treatment-related adverse events were diarrhea (85%), nausea (43%), rash (36%), vomiting (29%), and decreased appetite ( 25%).
Mobocertinib chemical structural formula (Source: medchemexpress.cn)
Dr. Christopher Arendt, head of the oncology treatment field at Takeda, said: "We are pleased that the FDA has recognized the therapeutic potential of mobocertinib for patients with NSCLC carrying the EGFR exon 20 insertion mutation. These patients urgently need effective treatment options. We are committed to developing innovative drugs for the treatment of refractory diseases. Mobocertinib ’s breakthrough drug qualification is an important step forward in our efforts to change the current standard of care for underserved people. "
Lung cancer is the leading cause of cancer death worldwide. Non-small cell lung cancer (NSCLC) is the most common type of lung cancer, accounting for about 85% of lung cancer cases. EGFR exon 20 insertion mutations account for only 1-2% of patients with NSCLC, and the prognosis is worse than other EGFR mutations. There is currently no treatment for exon 20 mutations. The benefits of these patients are limited.
Mobocertinib is a potent small molecule tyrosine kinase inhibitor (TKI) designed specifically to target EGFR and HER2 exon 20 insertion mutations. In 2019, the US FDA granted mobocertinib orphan drug qualification (ODD) for the treatment of lung cancer patients with HER2 mutations or EGFR mutations (including exon 20 insertion mutations).
The mobocertinib development project started in the NSCLC population and is expected to expand to other underserved populations of other tumor types. Mobocertinib is an investigational drug whose efficacy and safety have not been determined. (from Bioon.com）