AI Summary
In a multicentre, single-arm, phase 2 study, belzutifan demonstrated significant antitumour activity in patients with von Hippel-Lindau (VHL) disease-associated CNS haemangioblastomas. The treatment was effective for over 3 years, solidifying belzutifan as a viable systemic treatment for this patient population.
Belzutifan showed meaningful antitumour activity in VHL disease-associated CNS haemangioblastomas that was sustained for more than 3 years of treatment. These results continue to support belzutifan as a systemic treatment option for patients with VHL disease-related CNS haemangioblastomas.