Orphan drug status to tepotinib for NSCLC in JapanFebruary 6, 2020
The Japanese Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug designation (ODD) for tepotinib for patients with non-small cell lung cancer (NSCLC) harbouring MET gene alterations.
Tepotinib is an oral MET inhibitor that is designed to inhibit the oncogenic MET receptor signaling caused by MET gene alterations, including both MET exon 14 skipping alterations and MET amplifications, or MET protein overexpression. It has been designed to have a highly selective mechanism of action, with the potential to improve outcomes in aggressive tumours that have a poor prognosis and harbour these specific alterations.
lterations of the MET signaling pathway are found in various cancer types, including 3-5% of NSCLC cases, and correlate with aggressive tumour behaviour and poor clinical prognosis. Lung cancer is the most common type of cancer worldwide, with two million cases diagnosed annually.
Tepotinib is being investigated in the ongoing VISION study (NCT02864992), which showed preliminary efficacy in patients harbouring METex14 skipping alterations detected by liquid biopsy (LBx) or tissue biopsy (TBx) across different lines of treatment.
Tepotinib is also being investigated in the INSIGHT 2 study (NCT03940703) in combination with the tyrosine kinase inhibitor (TKI) osimertinib in epidermal growth factor receptor (EGFR) mutated, MET amplified, locally advanced or metastatic NSCLC having acquired resistance to prior EGFR TKI.