▎WuXiEdited by Kant Content Team

MSD announced today,The European Commission has granted conditional approval for the marketing of MSD's oral hypoxia-inducible factor 2α (HIF-2α) inhibitor Welireg (belzutifan) as a monotherapy for the following indications:：

• Adult patients with von Hippel-Lindau (VHL) disease who have localized renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET) that are not suitable for surgery and require treatment.

• Adult patients with advanced clear cell RCC whose disease has progressed after receiving two or more prior lines of therapy (including a PD-1 or PD-L1 inhibitor and at least two vascular endothelial growth factor-targeted therapies).

The press release pointed out,Welireg is the first HIF-2α inhibitor approved by the EU and also the first systemic therapeutic drug for treating VHL disease-related tumors.

The approvals for these two indications were based on the results of the LITESPARK-004 and LITESPARK-005 trials, respectively. The results of the LITESPARK-004 trial showed,In VHL disease-associated RCC patients (n=61), the objective response rate (ORR) for Welireg was 49% (95% CI, 36–62), with all responses being partial responses (PR). The median duration of response has not yet been reached in these patients, and 56% of responding patients maintained their response for at least 12 months.

In VHL disease-associated CNS hemangioblastoma patients (n=24), the ORR for Welireg was 63% (95% CI, 41–81). The median duration of response has not yet been reached in these patients, and among those who achieved a response, 73% maintained the response for at least 12 months.

In VHL disease-associated pNET patients (n=12), the ORR for Welireg was 83% (95% CI, 52–98), and the median duration of response had not yet been reached. Among the patients who achieved a response, 50% maintained the response for at least 12 months.

LITESPARK-005 trial results showed that Welireg reduced compared to the active control.Patients with clear cell RCCA 25% reduction in the risk of disease progression or death (HR=0.75, 95% CI, 0.63–0.90; p=0.0008).The ORR in the Welireg group was 22%, with a complete response rate of 3% and a PR rate of 19%; the corresponding value in the active control group was 4%, with no patients achieving complete response and a PR rate of 4%.

Welireg is the first approved HIF-2α inhibitor to be marketed., which has previously been approved in multiple countries and regions including the United States, the United Kingdom, and Canada. MSD is also continuously expanding its application potential, actively promoting the use of Welireg as a monotherapy or in combination with the PD-1 inhibitor pembrolizumab in RCC, endometrial cancer, esophageal cancer, liver cancer, prostate cancer, and rare cancers.

▲To learn more about the application of cutting-edge technologies in the biopharmaceutical industry, please long-press and scan the QR code above to access "WuXi Live Studio" and watch live discussions and exciting replays on related topics.