The therapy is the first approved for patients with tumors associated with von Hippel-Lindau disease.
Merck has selected the specialty pharmacy network Onco360 for Welireg (belzutifan), which the FDA recently approved to treat cancers associated with a rare genetic disorder called von Hippel-Lindau syndrome.
Welireg is a hypoxia-inducible factor inhibitor indicated for the treatment of adult patients with von Hippel-Lindau disease with renal cell carcinoma, central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors.
Merck executives said in a press release the company is working to optimize production of Welireg “to allow for a sustainable supply to meet anticipated U.S. demand,” and expects it to be available by early September.
The medication’s recommended dose is 120 mg once daily until disease progression.
Onco360 is headquartered in Louisville, Kentucky, and dispenses nationally through its network of URAC-, and ACHC-accredited oncology pharmacies.
Von Hippel-Lindau syndrome effects 1 in 36,000 people, according to the National Organization for Rare Disorders. The mean age of onset is 26 years, and 97% of people with a syndrome's genetic mutation have symptoms by the age of 65.
The approval of Welireg was based on Study OO4, an open-label trial in 61 patients. Nearly half of all patients with von Hippel Lindau-associated renal cell carcinoma, as well as the majority of patients with von Hippel-Lindau-associated central nervous system hemangioblastomas or pancreatic neuroendocrine tumors, experienced a reduction in their tumors. The overall response rate was 49%.