FDA Grants Tepotinib Breakthrough Therapy Designation

The FDA has granted Merck’s investigational new drug, tepotinib, a Breakthrough Therapy Designation in patients with metastatic non-small cell lung cancer (NSCLC) harboring MET exon 14 skipping alterations who progressed following platinum-based cancer therapy. 

Tepotinib is an investigational oral MET kinase inhibitor discovered in-house at Merck, and is designed to be highly potent, selective, and to inhibit the oncogenic signaling caused by MET gene alterations including both MET exon 14 skipping alterations and MET amplifications, or MET protein overexpression. 

The new Breakthrough Therapy Designation is based on data from the ongoing VISION study (NCT02864992).

Interim analysis from the trial was derived from 73 efficacy-evaluable patients with NSCLC with MET expon 14 skipping alterations identified by liquid biopsy (LBx) or tissue biopsy (TBx). Overall objective response rate in LBx-identified patients was 50.0% as measured by an independent review committee (IRC) and 55.3% as measured by investigators. 

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Overall median duration of response in LBx-identified patients was 12.4 months (IRC) and 17.1 months (investigators). In TBx-identified patients, the overall median duration of response was 15.7 months and 14.3 months, respectively. 

Treatment-related adverse events (TRAEs) reported in more than 10% of 87 patients evaluable for safety include peripheral edema, nausea, diarrhea, and increased blood creatinine. Other relevant TRAEs as listed in the release include increased lipase, fatigue, and vomiting. TRAEs reportedly led to permanent discontinuation in four patients-two patients suffering from peripheral edema, one patient suffering from interstitial lung disease, and one patient suffering from diarrhea and nausea.