FDA Approves Welireg for Several Cancers Associated With VHL Disease

The Food and Drug Administration (FDA) on Friday approved Welireg (belzutifan) for adults with von Hippel-Lindau (VHL) disease who require treatment for renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET).

The indication is for patients who do not require immediate surgery for their disease.

The FDA’s decision was based on the results of a clinical trial that evaluated the efficacy of the drug given at a once-daily dose of 120 milligrams (mg) until disease progression or unacceptable toxicity was reached. The trial consisted of 61 patients with VHL-associated RCC, as well as CNS hemangioblastomas and pNET.

The data demonstrated that treatment with Welireg induced an overall response rate (a percentage of patients with a partial or complete response to treatment) of 49% in patients with VHL-associated RCC. Those individuals with VHL-associated RCC who had a response were followed for a minimum of 18 months after the start of treatment. More than half of those patients (56%) experienced a duration of response (the length of time that a tumor continues to respond to treatment without the cancer growing or spreading) of at least 12 months and the median time to response was eight months.

Overall response rates for 24 patients with CNS hemangioblastomas and 12 patients with pNET were 63% and 83%, respectively. And response duration of at least 12 months occurred in 73% of patients with CNS hemangioblastomas and 50% of patients with pNET.

Common side effects that were reported in at least 20% of patients included low blood count levels, anemia, fatigue, kidney disease, headache, dizziness, increased glucose, and nausea. Severe side effects included anemia and low oxygen levels in the blood.

Read more: Life With von Hippel-Lindau Disease.

Check back later on what you need to know about this approval.

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