【FDA Approval Alert】Merck’s belzutifan (Welireg) Approved for Pheochromocytoma and Paraganglioma (May 14, 2025)
On May 14, 2025, the U.S. FDA approved belzutifan (Welireg) for patients aged 12 years and older with locally advanced, unresectable, or metastatic pheochromocytoma or paraganglioma (PPGL). This marks the first oral therapy approved for PPGL. Approval was based on the LITESPARK-015 trial (NCT04924075).
Disease Overview: Pheochromocytoma and Paraganglioma (PPGL)
PPGL are rare neuroendocrine tumors originating in adrenal or extra-adrenal tissues. Patients often present with hypertension and metabolic complications. Systemic therapy is required for unresectable disease.
Treatment Summary
- Drug Name: belzutifan (Welireg)
- Company: Merck & Co., Inc.
- Approval Date: May 14, 2025
- Indication: Locally advanced, unresectable, or metastatic PPGL
- Mechanism of Action: HIF-2α inhibition
- Pivotal Trial: LITESPARK-015 (NCT04924075)
- Key Efficacy: ORR 26% (Median DOR 20.4 months)
Patient-Friendly Summary
Welireg suppresses oxygen-sensing pathways in tumor cells, offering an oral treatment option for PPGL while potentially reducing antihypertensive medication burden.
M&A Information
- Subsidiary: Not applicable (Merck in-house development)
- Acquisition Date: Not applicable
- Development Stage at Acquisition: Not applicable
- Investor Information: Public Investors (NYSE: MRK)
Source
This approval information is based on data from Drugs@FDA (U.S. FDA) and AACR releases.
