USFDA grants priority review to Merck’s NDA for HIF-2α inhibitor Belzutifan

The US Food and Drug Administration (FDA) has granted priority review for Merck’s New Drug Application (NDA) for the hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor belzutifan.

A candidate from Merck’s oncology pipeline, Belzutifan is a novel investigational therapy for the potential treatment of patients suffering from von Hippel-Lindau (VHL) disease-associated renal cell carcinoma (RCC), which is not in need of immediate surgery.

The FDA has set September 15, 2021 as a Prescription Drug User Fee Act (PDUFA), or target action.

Merck Research Laboratories vice-president, clinical research, Dr. Scot Ebbinghaus said: “Von Hippel-Lindau disease is a rare genetic condition for which there is no systemic treatment option available and is associated with a high risk of cancer development in multiple organs. In fact, up to 70% of patients with VHL develop renal cell carcinoma during their lifetime. 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.”

Belzutifan (MK-6482) is a new, yet potent, selective inhibitor of HIF-2α. The VHL biology research that led to HIF-2α being discovered was awarded the Nobel Prize in Physiology or Medicine, in 2019.

The rare genetic disease of VHL affects one in 36,000 people, with 200,000 cases currently known worldwide. These patients are not only at risk for benign blood vessel tumours but also several types of cancers; one such cancer being renal cell carcinoma (RCC), which affects 70% of people.

The most common type of kidney cancer, RCC caused about 179,400 deaths in 2020.