Merck & Co has received a priority review from the US Food and Drug Administration (FDA) for its potential rare kidney cancer treatment belzutifan.
Belzutifan, an investigational hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor, is being investigated for the treatment of patients with von Hippel-Lindau (VHL) disease-associated renal cell carcinoma (RCC) who do not require immediate surgery.
VHL disease is a rare genetic disease which puts patients at risk of developing benign blood vessel tumours as well as several cancers, including RCC. Up to 70% of people with VHL disease develop RCC.
The new drug application (NDA) for belzutifan is based on data from the Phase II Study-004 trial.
In this trial, belzutifan demonstrated a confirmed overall response rate of 36.1% in patients with VHL disease-associated RCC. Secondary endpoints include disease control rate, duration of response, time to response, progression-free survival, time to surgery and safety.
"This priority review validates the important progress we have made to expand and diversify Merck's oncology pipeline with innovative, new therapeutic approaches. We look forward to working closely with the FDA to bring belzutifan to patients in need,” said Scot Ebbinghaus, vice president, clinical research, Merck Research Laboratories.
Merck & Co is also studying belzutifan in advanced RCC and other tumour types as part of its clinical programme for the HIF-2α inhibitor.
This includes Phase III trials evaluating belzutifan as monotherapy and as part of a combination regimen in previously treated patients, and as part of a combination regimen for the first-line treatment of advanced clear cell RCC.