FDA approval expands Welireg to patients with renal cell carcinoma

Welireg was originally approved by the FDA in 2021 for a rare genetic condition. The latest approval allows the drug to be used in patients with advanced RCC that have previously received checkpoint inhibitors targeting programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) and blockers of vascular endothelial growth factor tyrosine kinase inhibitor (VEGF-TKI).

RCC is the most common form of kidney cancer and advanced forms are historically hard to treat​ with traditional chemotherapy. The arrival of checkpoint inhibitors and VEGF-TKI blockers is providing ever more options for patients with RCC.

However, more options are needed for patients whose cancer progresses after receiving these treatments, said a public release from MSD.

The approval decision was based on results from phase 3 trial named LITESPARK-005, where belzutifan boosted progression-free survival compared to the renal cell cancer therapy everolimus in patients whose cancer had progressed following treatment with a PD-1 or PD-L1 checkpoint inhibitor and a VEGF receptor targeted therapy.

“This approval of Welireg marks the first new therapeutic class available for eligible patients with advanced RCC in nearly a decade,” stated Marjorie Green, senior vice president and head of late-stage oncology, global clinical development, Merck Research Laboratories.

Welireg is an oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor. MSD first got hold of the compound when it acquired the company Peloton Therapeutics in 2019 for up $2 billion.