FDA nod for belzutifan in cancers linked with VHL

The US Food and Drug Administration on Friday approved Welireg (belzutifan), a hypoxia-inducible factor inhibitor for adult patients with von Hippel-Lindau (VHL) – a rare kidney disease - who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET), not requiring immediate surgery. The approval was granted to US pharma giant Merck & Co (NYSE: MRK).

The FDA’s decision comes well ahead of the expected September 15 action date, and it includes a surprisingly broader label beyond just renal cell carcinoma patients, Cantor Fitzgerald analysts said in a note to clients Friday. Evaluate Pharma has previously forecast sales of $386 million for Welireg by 2026.

“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, adding: “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.”