Story perspectives

Breakthrough Drug Belzutifan Shrinks Tumors in Cancer Trial

10/18/2025

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Summary

Story summary

The University of Texas MD Anderson Cancer Center led a Phase II trial showing belzutifan, an oral HIF-2? inhibitor, shrinks tumors and improves symptoms in 72 patients with advanced pheochromocytoma and paraganglioma (PPGL), achieving a 26% objective response rate and benefits longer than 20 months.
All participants had exhausted standard treatments before enrolling.
In May 2025, the U.S. FDA approved belzutifan as the first systemic therapy for PPGL.

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Story summary

The University of Texas MD Anderson Cancer Center led a Phase II trial showing belzutifan, an oral HIF-2? inhibitor, shrinks tumors and improves symptoms in 72 patients with advanced pheochromocytoma and paraganglioma (PPGL), achieving a 26% objective response rate and benefits longer than 20 months.
All participants had exhausted standard treatments before enrolling.
In May 2025, the U.S. FDA approved belzutifan as the first systemic therapy for PPGL.