Pexidartinib met its primary endpoint of tumor response as measured by tumor size reduction in patients with symptomatic tenosynovial giant cell tumor (TGCT) where surgical resection is contraindicated, according to results from the phase III ENLIVEN trial.
The most common pexidartinib-related all-grade adverse events (AEs) were changes in hair color (74%), fatigue (65%), nausea (39%), dysgeusia (26%), and periorbital edema (26%). The most frequent grade ≥3 AEs were hyponatremia (9%), elevated aspartate aminotransferase or alanine aminotransferase level or both (9%), fatigue (4%), diarrhea (4%), anemia (4%), and neutropenia 4%.
Tap WD, Wainberg ZA, Anthony SP, et al. Structure-Guided Blockade of CSF1R Kinase in Tenosynovial Giant-Cell Tumor. N Engl J Med. 2015;373:428-437.
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