Merck grabs first FDA nod for rare VHL tumors

Aug. 16, 2021

The U.S. FDA has approved Merck & Co's (MSD) blockbuster hopeful, Welireg, for patients with the rare disorder known as von Hippel-Lindau (VHL) disease — making the drug the first systemic therapy approved to treat patients diagnosed with certain types of VHL-associated tumors.

VHL is a rare genetic disease that causes tumors and cysts to grow throughout the body. It has an estimated incidence of 10,000 people in the U.S.

Welireg's approval was based on data from an open-label trial in 61 patients with VHL-associated renal cell carcinoma – some of whom also had other tumors. The FDA nod, which came well ahead of the expected September 15 action date, goes beyond just renal cell carcinoma patients, giving the drug the green light for the treatment of patients with VHL disease who require therapy for associated renal cell carcinoma, central nervous system hemangioblastomas or pancreatic neuroendocrine tumors.

Merck picked up Welireg — an oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor —through its 2019 $2.2 billion takeover of Peloton Therapeutic.

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