The U.S. Food and Drug Administration has approved the use of Merck's cancer drug, Welireg (belzutifan), for treating two rare types of adrenal gland tumors: pheochromocytoma and paraganglioma (PPGL). This marks Welireg as the first oral treatment approved for these tumors, providing a new and more convenient option for patients with these conditions.
The expanded approval specifically targets patients aged 12 and older with metastatic or inoperable PPGL, according to Reuters. Clinical trials have shown that Welireg achieves a 20.6% objective response rate in treating these tumors, potentially improving patient care where previously, treatment options were limited primarily to surgery or palliative care.
Before this approval, systemic options for managing PPGL were scarce. This new indication for Welireg aligns with its already existing approvals for cancers associated with Von Hippel-Lindau Disease and renal cell carcinoma, which contributed to $509 million in sales for Merck last year. This expansion signifies a meaningful step forward in addressing significant unmet medical needs for these rare conditions.