On 15 February 2024, the US Food and Drug Administration (FDA) granted traditional approval to tepotinib (Tepmetko, EMD Serono, Inc.) for adult patients with metastatic non-small cell lung cancer (NSCLC) harbouring mesenchymal-epithelial transition (MET) exon 14 skipping alterations.
Tepotinib was previously granted accelerated approval for this indication on 3rd February 2021, based on initial overall response rate (ORR) and duration of response (DoR) in the VISION study (NCT02864992), a multicentre, non-randomised, open-label, multicohort study. The conversion to traditional approval was based on an additional 161 patients and an added 28 months of follow-up time to assess DoR.
Efficacy was demonstrated in a total of 313 patients with metastatic NSCLC harbouring MET exon skipping alterations. Patients received tepotinib 450 mg once daily until 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% confidence interval [CI] 49, 65), with 40% of responders having a DoR ≥12 months. Among 149 previously treated patients, ORR was 45% (95% CI 37, 53), with 36% of responders having a DoR ≥12 months.
The most common adverse reactions (≥20%) were oedema, nausea, fatigue, musculoskeletal pain, diarrhoea, dyspnoea, decreased appetite, and rash.
The recommended tepotinib dose is 450 mg orally once daily with food.
This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.
This application was granted breakthrough designation and orphan drug designation.
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.
For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact FDA’s Oncology Center of Excellence Project Facilitate.
