VHL Historic Treatment Breakthrough

FDA Approval of Breakthrough Treatment Brings Welcome Hope

The US Food and Drug Administration (FDA) has approved the first-ever pharmaceutical treatment for patients with Von Hippel-Lindau (VHL) disease, a genetic condition putting patients at risk of developing several cancers and other types of tumors. Beluztifan (Welireg, Merck), a novel HIF-2α inhibitor, was granted Breakthrough Therapy designation in March 2021 and approved by the FDA on August 13, 2021. Its development can be traced through Nobel Prize-winning research and is in further trials for patients with kidney, pancreatic, and other cancers.
Von Hippel-Lindau disease is a genetic disorder characterized by tumours that can occur at any time throughout a patient’s lifetime in up to ten different areas of the body, including the kidneys, brain, spine, pancreas, adrenal glands, eyes, and ears. Until now, the only way to treat VHL was with regular surveillance (MRIs and other tests), followed by timely surgical removal of tumours before they can cause permanent damage. Most VHL patients face multiple, high-risk and expensive surgeries over their
lifetimes. Systemic therapy has long been the hope of patients and their families.

Click here to view our exclusive video on Von Hippel-Lindau with Professor Dr Tze Min Wah and patient Alan Speight