On February 15, 2024, the U.S. Food and Drug Administration (FDA) granted traditional approval to tepotinib (Tepmetko®) for adult patients with metastatic non-small cell lung cancer (NSCLC) harboring mesenchymal-epithelial transition (MET) exon 14–skipping alterations.

FDA update

FDA previously granted tepotinib accelerated approval for the indication on February 3, 2021, based on initial overall response rate (ORR) and duration of response (DOR) in the VISION trial (NCT02864992), a multicenter, nonrandomized, open-label, multicohort study. The conversion to traditional approval was based on an additional 161 patients and another 28 months of follow-up to assess DOR.

Efficacy was demonstrated in 313 patients with metastatic NSCLC harboring MET exon-skipping alterations. Patients received tepotinib 450 mg once daily until they experienced disease progression or unacceptable toxicity.

The primary efficacy measures were ORR and DOR, determined by a blinded independent review committee. Among 164 treatment-naïve patients, ORR was 57% (95% CI = 49, 65), with 40%  having a DOR of at least 12 months. Among 149 previously treated patients, ORR was 45% (95% CI = 37, 53), with 36% having a DOR of at least 12 months.

The most common adverse reactions reported in at least 20% of patients in the VISION trial were edema, nausea, fatigue, musculoskeletal pain, diarrhea, dyspnea, decreased appetite, and rash.

The recommended tepotinib dose is 450 mg orally once daily with food. View the full prescribing information here.

The applicant used the Assessment Aid, a voluntary submission to facilitate FDA’s assessment. The application was granted breakthrough designation and orphan drug designation. FDA-expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions—Drugs and Biologics.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 800-FDA-1088.

For assistance with single-patient applications for investigational oncology products, contact the Oncology Center of Excellence’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.