Merck receives FDA nod for Welireg
The Food and Drug Administration has approved Merck's Welireg (belzutifan), the first treatment for patients with von Hippel-Lindau disease, a genetic condition putting patients at risk of developing several cancers and other types of tumors.
"For VHL patients, the long-held hope and belief that this battle can be won is now a reality. Thank you to the patients that have participated in clinical trials and researchers who have worked tirelessly for this breakthrough. It is because of your hard work and sacrifice that VHL-related surgeries may now be a thing of the past. We are grateful for this evidence that proves that we must remain persistent in our pursuit of Curing Cancer through VHL," said Chandra Clark, executive director of the VHL Alliance.
The medication is approved for adult patients with von Hippel-Lindau disease who require therapy associated with renal cell carcinoma, central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors, not requiring immediate surgery.
“Welireg is the first and only approved systemic therapy for patients with certain types of VHL-associated tumors, representing an important new treatment option for patients affected by this rare condition,” said Dr. Scot Ebbinghaus, vice president, clinical research, Merck Research Laboratories. “Today’s approval of Welireg is a significant milestone and is a testament to Merck’s commitment to bring forward innovative new treatment options for more patients.”
"The approval of belzutifan for treatment of VHL-related renal cancers, hemangioblastomas, and neuroendocrine tumors of the pancreas is a game-changer in the treatment of VHL patients," said Othon Iliopoulos, director of the VHL Comprehensive Clinical Care Center at Massachusetts General Hospital and Harvard Medical School, associate professor of Medicine at Harvard Medical School.
Iliopoulos continued, "We now have a well-tolerated pill with the efficacy to treat a disease for which surgery was previously the only option. The resulting impact on patient quality of life will be immense. I would now like to see us initiate clinical trials designed to help us understand the optimal time to start the medication and to determine the appropriate length of therapy."