FDA Approves Targeted Treatment for Lung Cancer Along with Companion Diagnostic

The U.S. Food and Drug Administration (FDA) approved Tabrecta™ (capmatinib) for adults with metastatic non-small cell lung cancer (NSCLC) with the MET exon 14 skipping mutations, making it the first FDA approval to target these mutations. The FDA also approved the FoundationOne CDx assay as a companion diagnostic to determine MET exon 14 skipping mutations.

The decision was based on a phase II clinical trial in which 97 patients received capmatinib 400 mg orally twice daily until disease progression or unacceptable toxicity. The population included 28 patients who had never undergone treatment for NSCLC and 69 previously treated patients.

Response observed in treatment-naïve and previously treated patients

The overall response rate (ORR; primary endpoint) for untreated patients with 68%, with 4% experiencing a complete response and 64% experiencing a partial response (PR). The ORR for previously treated patients was 41%, all of whom experienced a PR. Among those who responded to capmatinib who had not undergone previous treatment for NSCLC, 47% had a duration of response lasting 12 months or longer compared with 32.1% of respondents who had been previously treated.

Common adverse events (AEs) associated with capmatinib include peripheral edema, nausea, fatigue, vomiting, dyspnea, and decreased appetite.

Capmatinib may cause serious AEs, including interstitial lung disease or pneumonitis. Capmatinib may also cause hepatotoxicity, so patients’ liver function should be tested prior to starting and during treatment.