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FDA Decision Alerts July 23, 2020

CDER-Approved NDA for TABRECTA™ (capmatinib hydrochloride)

Nick Honig and Tony Louder
Contributing writers, Aetion

On May 6, 2020, the FDA approved Novartis’s TABRECTA™ (capmatinib hydrochloride) for treatment of metastatic non-small cell lung cancer with mutations leading to MET exon 14 skipping (advanced MET-mutated NSCLC). The FDA granted Orphan Drug and Breakthrough Therapy designations for capmatinib hydrochloride. Key findings from the FDA’s Multi-Discipline Review: The applicant demonstrated efficacy of capmatinib hydrochloride for treatment of advanced MET-mutated NSCLC in a multicenter, non-randomized, open-label, multi-cohort study, Study A2201 (GEOMETRY Mono-1). In addition, the sponsor submitted Study X2401, a global retrospective chart analysis to provide the natural history of advanced MET-dysregulated NSCLC. Purpose ‍The efficacy review focused primarily on Study A02201, a Phase 2 trial with no placebo arm. The review also considered evidence from Study X2102, a Phase 1, open-label dose-escalation trial. The sponsor also included Study X2401, a global retrospective chart study, to describe the natural history of advanced MET-mutated NSCLC. What was done ‍In Study X2401, the applicant completed a chart review of 157 patients with advanced MET-mutated NSCLC. The applicant did not include the patient-level data in its submission. Study X2401 showed advanced MET-mutated NSCLC is more common in older patients, and demonstrated that gender, smoking history, and tumor histology have no bearing on the disease’s occurrence. The analysis found the MET mutation is mutually exclusive from other molecular drivers. Outcome ‍The reviewer stated Study X2401 provided “clinically significant” information that capmatinib hydrochloride “fills an unmet medical need.” The reviewer also found the natural history study’s findings with respect to immunotherapy “further strengthen the clinical relevance of the efficacy of capmatinib irrespective of the line of treatment.” The reviewer noted “while FDA considers data from… Study X2401 to be supportive, Novartis did not submit this data, and thus FDA cannot independently verify these results.” ‍

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