Evaluation of the primary endpoint showed positive results for Welireg in advanced renal cell carcinoma treatment

A randomized, open-label phase 3 trial (LITESPARK-005, ClinicalTrials.gov, NCT04195750) investigated the efficacy of Welireg (Belzutifan) in comparison to Everolimus for the treatment of advanced renal cell carcinoma (RCC) that has progressed after prior treatment with PD-1/L1 and VEGF-TKI therapies, in sequence or in combination. The active substance in Welireg is Belzutifan which is chemically a hypoxia-inducible-2 alpha (HIF-2α) inhibitor.

Welireg is a prescription medicine indicated in the treatment of von Hippel-Lindau (VHL) disease in patients who require treatment for associated renal cell carcinoma (RCC), spinal cord or brain tumors (hemangioblastomas), pancreatic neuroendocrine tumors (pNET) and do not require surgery right away.

The trial enrolled patients (N=746) who were randomized to receive Welireg (120 mg oral once daily) or Everolimus (10 mg oral once daily).

• The primary endpoint evaluation showed positive results as Welireg has proven to show improvement in progression-free survival.
• Welireg has shown improvement in progression-free survival. A statistically significant improvement in the key secondary endpoint of objective response rate (ORR) was also observed.
• A trend toward improvement in overall survival (OS), a dual primary endpoint was also seen however, this result was not statistically significant.
• These results were consistent and had not been reported earlier. The safety and effectiveness of WELIREG in pediatric patients under 18 years of age have not been established.

Welireg is the first HIF-2α inhibitor approved in the U.S. and is currently approved in the U.S., Great Britain, Canada, and other countries for the treatment of von Hippel-Lindau (VHL) disease in patients who require therapy for associated RCC, central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors and not requiring immediate surgery.