FDA Approves First Targeted Therapy for Certain Patients with NSCLC


Non-small cell lung cancer is a the most common type of lung cancer with up to 90% of all lung carcinomas falling into the non-small cell category.

Officials with the FDA have approved capmatinib (Tabrecta, Novartis Pharmaceuticals) for the treatment of adult patients with non-small cell lung cancer (NSCLC) that has spread to other parts of the body. Capmatinib is the first FDA-approved therapy to treat NSCLC with specific mutations (those that lead to mesenchymal-epithelial transition or MET exon 14 skipping), according to the agency.

NSCLC is a the most common type of lung cancer with up to 90% of all lung carcinomas falling into the non-small cell category. With this form of the disease, there is a high likelihood that the cancer cells will spread from the lungs to other organs and body parts, according to the FDA.

Cancer metastasis consists of a sequential series of events, and MET exon 14 skipping is recognized as a critical event for metastasis of carcinomas. Mutations leading to MET exon 14 skipping are found in 3% to 4% of patients with lung cancer.

“Lung cancer is increasingly being divided into multiple subsets of molecularly defined populations with drugs being developed to target these specific groups,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, in a prepared statement.

In addition to the drug approval, officials with the FDA have approved Foundation Medicine’s FoundationOne CDx assay (F1CDx) as a companion diagnostic for capmatinib. Most patients had tumor samples that were tested for mutations that lead to MET exon 14 skipping using local tests and confirmed with the F1CDx, which is a next-generation sequencing based in vitro diagnostic device that is capable of detecting several mutations, including mutations that lead to MET exon 14 skipping.

Capmatinib is a kinase inhibitor that blocks a key enzyme, which results in helping to stop the tumor cells from growing. The approval of capmatinib was based on the results of a clinical trial involving patients with NSCLC with mutations that lead to MET exon 14 skipping, epidermal growth factor receptor (EGFR) wild-type and anaplastic lymphoma kinase (ALK) negative status, and at least 1 measurable lesion.

During the clinical trial, participants received capmatinib 400 mg orally twice daily until disease progression or unacceptable toxicity. The major efficacy outcome measure was overall response rate (ORR), which reflects the percentage of participants who had a certain amount of tumor shrinkage. An additional efficacy outcome measure was duration of response (DOR).

The efficacy population included 28 patients who had never undergone treatment for NSCLC and 69 previously treated patients. The ORR for the 28 participants was 68%, with 4% having a complete response, and 64% having a partial response. The ORR for the 69 participants was 41%, with all having a partial response. Of the responding participants who had never undergone treatment for NSCLC, 47% had a duration of response lasting 12 months or longer compared with 32.1% of the responding participants who had been previously treated.

Common adverse effects (AEs) for patients taking capmatinib are leg swelling, nausea, fatigue, vomiting, shortness of breath, and decreased appetite.

Capmatinib may cause serious AEs, including interstitial lung disease and pneumonitis. This drug should be permanently discontinued in patients with these AEs. Capmatinib may also cause hepatotoxicity, and health care professionals should monitor a patient’s liver function tests prior to starting and when taking this drug. If a patient experiences hepatotoxicity, Tabrecta should be withheld, dose reduced, or permanently discontinued.

Based on a clear positive signal for phototoxicity in laboratory studies in cells, patients may be more sensitive to sunlight and should be advised to take precautions to cover their skin, use sunscreen, and not to tan while taking capmatinib.

Capmatinib may cause harm to a developing fetus or newborn baby. Health care professionals should advise pregnant women of this risk and should advise females of reproductive potential and male patients with female partners of reproductive potential to use effective contraception during treatment with this drug and for 1 week after the last dose.

Capmatinib was approved under the Accelerated Approval pathway. The FDA previously granted the application for this drug Breakthrough Therapy designation and Priority Review designation. Capmatinib also received Orphan Drug designation.


FDA Approves First Targeted Therapy to Treat Aggressive Form of Lung Cancer [news release]. Silver Spring, MD; May 6, 2020: FDA website. https://www.fda.gov/news-events/press-announcements/fda-approves-first-targeted-therapy-treat-aggressive-form-lung-cancer. Accessed May 7, 2020

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