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Up to 15,000 people in Europe are living with VHL, a rare tumour-causing genetic disease
Welireg has been authorised for use in adults with VHL disease who require therapy for associated, localised RCC, central nervous system haemangioblastomas, or pancreatic neuroendocrine tumours, and for whom localised procedures are unsuitable.
The drug has also been approved to treat adults with advanced clear cell RCC that has progressed following at least two lines of therapy that included a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor, and two or more vascular endothelial growth factor (VEGF) targeted therapies.
Up to 15,000 people in Europe are living with VHL disease, which occurs when a mutation in the VHL gene causes cells to grow abnormally. This can lead to cysts or tumours developing in different parts of the body, including the kidneys and pancreas, that can impair function or become cancerous.
RCC is far more common, with more than 130,000 new cases of the kidney cancer diagnosed in Europe in 2020 alone. The disease disproportionately affects men and approximately 30% of patients are diagnosed at an advanced stage.
Merck gained access to Welireg in 2019 through its $1.1bn buyout of Peloton Therapeutics. The drug is taken orally and is designed to inhibit the transcription factor hypoxia-inducible factor 2 alpha (HIF-2α), which regulates cellular proliferation, angiogenesis and tumour growth.
The EC’s decision on the two indications follows a recent recommendation from the European Medicines Agency’s human medicines committee and was supported by positive results from the phase 2 LITESPARK-004 trial in VHL disease and the phase 3 LITESPARK-005 study in RCC.
Marjorie Green, senior vice president and head of oncology, global clinical development, Merck Research Laboratories, said: “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 localised procedures are unsuitable, and offers a new option for adult patients with advanced clear cell RCC that progressed following a PD-1 or PD-L1 inhibitor and at least two VEGF targeted therapies.
“This is an important moment, and we are pleased that Welireg, a first-in-class HIF-2α inhibitor, can now potentially help these patients in need.”
