Belzutifan for Advanced Pheochromocytoma or Paraganglioma
Authors' conclusion
Belzutifan showed antitumor activity with durable responses in participants with advanced pheochromocytoma or paraganglioma.
Study context
- Population
- Locally advanced or metastatic pheochromocytoma or paraganglioma not amenable to surgery or curative-intent treatment
- Participants
- 72 participants
- Outcome
- Confirmed objective response (complete or partial response) by blinded independent central review
Methodological detailsphase 2, international
- Phase 2
- International
Main finding
As reported in the abstract
Confirmed objective response (complete or partial response), blinded independent central review · median follow-up 30.2 months (range, 23.3 to 37.6)
Participants with confirmed objective response
26%
95% CI 17 to 38
Further findings
Disease control · median follow-up 30.2 months
- Participants with disease control
- 85% (95% CI, 74 to 92)
Duration of response
- Median duration of response
- 20.4 months (95% CI, 8.3 to not reached)
PFS (blinded independent central review)
- Median PFS
- 22.3 months (95% CI, 13.8 to not reached)
Overall survival · 24 months
- Overall survival
- 76%
Reduction ≥50% in total daily dose of ≥1 antihypertensive medication for ≥6 months
- Participants with reduction, among 60 receiving antihypertensives
- 19 (32%)
Safety
Treatment-related adverse events
- Treatment-related adverse events
- 71 participants (99%)
- Grade 3 anemia
- 22%
Treatment-related serious adverse events
- Treatment-related serious adverse events
- 8 participants (11%)
Original publication
Belzutifan for Advanced Pheochromocytoma or Paraganglioma. N Engl J Med. Published 18 October 2025. doi:10.
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