In the phase 2 LITESPARK-015 trial, Belzutifan demonstrated antitumor activity in advanced pheochromocytoma or paraganglioma with an objective response rate of 26%. The responses were durable with a median duration of response of 20.4 months. The safety profile was manageable with serious adverse events observed in 11% of participants.
Study
|
Phase 2, international, single-group trial [LITESPARK-015, NCT04924075] |
| Locally advanced or metastatic pheochromocytoma or paraganglioma not amenable to surgery or curative-intent treatment |
| Belzutifan 120 mg once daily (n=72) until progression or unacceptable toxic effects
|
Efficacy
|
ORR: 26% [17-38] |
| mDOR: 20.4 mos [8.3-NR] |
| mPFS: 22.3 mos [13.8-NR] |
| 2-yr OS: 76%
|
Safety
|
Grade >=3 AE: anemia (22%), fatigue (4%), dyspnea (0%) |
| Serious AEs: 11% |
| Treatment discontinuation due to AEs: 3%
|
N Engl J Med. Published online 2025 Oct 18.
http://doi.org/10.1056/NEJMoa2504964
Reviewed by Ulas D. Bayraktar, MD on Nov 14, 2025
