HQ Team
February 18, 2025: Merck and Co’s drug, for treating adult patients with kidney and pancreatic cancer, tumours and who had prior treatment, has been “conditionally” approved by the European Commission, according to a company statement.
The drug Welireg treats adult patients with Von Hippel-Lindau (VHL) disease who require therapy for associated, localised renal cell carcinoma, central nervous system hemangioblastomas, or pancreatic neuroendocrine tumours for whom local procedures are unsuitable.
Von Hippel-Lindau disease, also known as Von Hippel-Lindau syndrome, is a rare genetic disorder impacting multiple systems in the body. It is characterized by the development of cysts and both benign and malignant tumours in various organs.
Hemangioblastomas are rare, benign tumours primarily found in the central nervous system, particularly in the cerebellum, brainstem, and spinal cord.
All 27 EU member states
They are characterised by their highly vascular nature and are classified as World Health Organization grade 1 tumours due to their low malignancy potential.
The EC approval allows marketing of Welireg for these indications in all 27 EU member states, as well as Iceland, Liechtenstein and Norway.
Welireg (belzutifan) is mainly meant to treat adult patients with advanced cell kidney cancer after two or more lines of therapy, according to the statement.
The EC approval follows a positive recommendation from the Committee for Medicinal Products for Human Use adopted in December 2024.
Valid for a year
“The approval of WELIREG in the EU introduces the first and only systemic treatment option for adult patients with certain VHL disease-associated tumours for whom localized procedures are unsuitable,” said Dr Marjorie Green, senior vice president and head of oncology, global clinical development, Merck Research Laboratories.
“It offers a new option for adult patients with advanced clear cell renal cell carcinoma that progressed following a PD-1 or PD-L1 inhibitor and at least two VEGF targeted therapies,” she said.
The conditional approval of Welireg will be valid for one year, subject to yearly renewal, pending additional clinical data and another ongoing mid-stage trial of the drug in patients with certain VHL disease-associated tumours.
“Timing for commercial availability of Welireg in individual EU countries will depend on multiple factors, including the completion of national reimbursement procedures,” according to the statement.
Renal cell carcinoma
Von Hippel-Lindau disease impacts an estimated 200,000 people worldwide and an estimated 10,000 to 15,000 people in Europe.
Patients with VHL disease are at risk for recurrent, benign blood vessel tumours as well as some cancerous ones. One of the most commonly occurring tumors is renal cell carcinoma, a form of kidney cancer, which occurs in about 70% of patients with VHL disease.
Renal cell cancer is a disease in which malignant cells are found in the lining of tubules in the kidney. There are two kidneys, one on each side of the backbone, above the waist. Tiny tubules in the kidneys filter and clean the blood.
