FDA grants Breakthrough Therapy Designation to HIF-2α Inhibitor MK-6482 for treatment of certain patients with Von Hippel-Lindau (VHL) disease-associated renal cell carcinoma (RCC)

This investigational, selective, oral HIF-2α inhibitor is currently being evaluated in a phase 3 trial in advanced RCC, a phase 2 trial in VHL-associated RCC and a phase 1/2 dose-escalation and dose-expansion trial in advanced solid tumours, including advanced RCC

SPS commentary:

Von Hippel-Lindau disease is a rare genetic disorder that affects one in 36,000 people. Proteins known as hypoxia-inducible factors, including HIF-2α, can accumulate in patients when VHL, a tumor-suppressor protein, is inactivated. The accumulation of HIF-2α can lead to the formation of both benign and malignant tumours. This inactivation of VHL has been observed in more than 90% of clear cell RCC tumours. As many as 60% of people with VHL disease develop RCC.


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