The take-at-home tablet will be available on the NHS in England and Wales to treat von Hippel-Lindau (VHL) disease in adults who require therapy for VHL-associated renal cell carcinomas, central nervous system haemangioblastomas or pancreatic neuroendocrine tumours, and for whom localised procedures are unsuitable or undesirable.
Affecting approximately one in every 70,000 people, VHL disease occurs when a mutation in the VHL gene causes cells to grow abnormally. This can lead to cysts or tumours developing in different parts of the body, including the kidneys, brain and pancreas that can impair function or become cancerous…
