FDA approves new drug for advanced lung cancer, first agent specific for patients with MET gene abnormalities

UCLA researcher, one of the lead investigators, presented data from clinical trial leading to drug approval at the American Association for Cancer Research Annual Meeting

The U.S. Food & Drug Administration has approved a new drug to treat patients with a type of advanced lung cancer that tends to have a poor prognosis. The drug, capmatinib, is the first FDA-approved therapy to target abnormalities in the MET gene, which accounts for up to 4% of non-small-cell lung carcinoma cases. This is the most common form of lung cancer. The drug can be used as a first-line therapy or in previously treated patients with locally advanced or metastatic MET exon 14 skipping mutated non-small-cell lung carcinoma. This is a group that has not typically done well with standard therapies such as chemotherapy or immunotherapy.

MET has been studied as a potential contributor to cancer for decades. However, MET exon 14 skipping mutations are a newer discovery. This is because researchers typically look for mutations in the portion of genes that directly generate a protein, but this is not the location of the mutations of relevance for capmatinib. The mutations that are effectively treated by capmatinib result in a portion of the MET protein important for its destruction being “left out.”

“Patients with MET exon 14 skipping mutations now have access to a drug targeting the specific abnormality that drives their tumor, and one that would be expected to improve their clinical outcomes as compared to standard therapy,” said one of the lead investigators, Edward Garon, MD, director of the Signal Transduction and Therapeutics Program at the UCLA Jonsson Comprehensive Cancer Center. “Although 4% of a disease may seem like a small population, as there are approximately 200,000 cases of lung cancer annually in the United States, this is a substantial number of patients. We have now shown that an agent targeting MET leads to better results than would be expected with standard treatments.”

Dr. Garon presented the data from the clinical trial that led to the FDA approval at the American Association for Cancer Research Annual Meeting on April 27. The meeting utilized a virtual format this year to facilitate presentation of important scientific data in an appropriate setting in light of the COVID-19 pandemic.

The team found that patients demonstrated substantial tumor shrinkage in over 2/3 of the first-line therapy patients and over 40% of the patients who were previously treated with other therapies.

“After many years of research related to the MET gene in the lung cancer community, it is gratifying to see that this research can now lead to better outcomes among patients with cancer,” said Garon, who is an associate professor of medicine at the David Geffen School of Medicine at UCLA.

Developed by Novartis, capmatinib was also found to substantially shrink tumors that spread to the brain in seven of the 13 evaluable patients who had brain metastases. Four of those patients saw their brain lesions completely disappear. This result is of particular interest, as brain metastases frequently pose a difficult issue for lung cancer patients.