FDA Approves First Targeted Therapy for Non-Small Cell Lung Cancer

The FDA has approved capmatinib (Tabrecta, Novartis) for the treatment of non-small cell lung cancer (NSCLC) that has spread to other parts of the body, along with its companion diagnostic FoundationOne CDx assay (F1CDx), according to a news release.

Capmatinib is a kinase inhibitor, which aids in slowing the growth of tumor cells. The treatment was approved through the Accelerated Approval pathway to Novartis Pharmaceuticals, while the F1CDx companion diagnostic approval was given to Foundation Medicine, Inc.

NSCLC is the most common type of lung cancer; up to 90% of all lung carcinomas are a member of the non-small cell category. NSCLC has a high risk of cancer cells spreading from the lungs to other organs and regions of the body.

“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 statement. “Tabrecta is the first approval specifically for the treatment of patients with non-small cell lung cancer whose tumors have mutations that lead to MET exon 14 skipping. This patient population now has an option for a targeted therapy, which they didn’t have prior to today.”

Approval of capmatinib was based on a clinical trial of 97 patients with NSCLC with mutations that led to MET exon 14 skipping, epidermal growth factor receptor (EGFR) wild-type and anaplastic lymphoma kinase (ALK) negative status, and 1 or more measurable lesions. The study evaluated overall response rate (ORR) in participants, meaning the amount of tumor shrinkage, as well as the duration of response (DOR). Twenty-eight participants had never undergone NSCLC treatment, whereas 69 had been previously treated for the disease.

The trial found an ORR of 68% for the previously untreated patients; 4% of the population had a complete response, whereas another 64% demonstrated a partial response. In the previously treated population, the ORR was 41%, and all patients in this arm showed a partial response.

The most frequent adverse effects for capmatinib are peripheral edema (leg swelling), nausea, fatigue, vomiting, dyspnea (shortness of breath) and lack of appetite.

“In the face of the COVID-19 pandemic, our regular work on reviewing treatments for patients with cancer is moving forward,” said Pazdur. “The impact may be hardest on those with acute or chronic medical conditions and those with weakened immune systems, such as that caused by cancer and some forms of cancer treatment. We are working to address critical issues for patients with cancer and their health care providers and continuing to expedite oncology product development in this critical time.”

References:

1. FDA Approves First Targeted Therapy to Treat Aggressive Form of Lung Cancer. News Release. FDA; May 6, 2020. Accessed May 6, 2020. https://www.fda.gov/news-events/press-announcements/fda-approves-first-targeted-therapy-treat-aggressive-form-lung-cancer.