Durable responses to belzutifan in patients with VHL disease

Abstract

The HIF2α inhibitor belzutifan has shown promising activity in patients with tumours arising in the context of Von Hippel–Lindau disease (VHL), an autosomal-dominant cancer predisposition syndrome arising from the deletion or inactivation of VHL. Now, long-term follow-up data from the phase II LITESPARK-004 trial demonstrate the durable activity of this agent as monotherapy in this population.

A total of 61 patients with VHL and renal cell carcinoma (RCC) received belzutifan until disease progression, discontinuation owing to toxicities, or study withdrawal. Objective response rate (ORR) significantly exceeding 30% was the primary end point. This end point was met, with an ORR of 49% reported at a median follow-up duration of 21.8 months.