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The CSF1R blocker pexidartinib shrank tenosynovial giant cell tumours in about four in ten patients against none on placebo, becoming the first approved drug for this joint tumour, with a liver-safety programme attached.
ENLIVEN randomised 120 patients with symptomatic, advanced tenosynovial giant cell tumour in whom surgery would cause worsening functional limitation or severe morbidity to pexidartinib or placebo for 24 weeks. The RECIST response rate at week 25 was 39 percent with pexidartinib versus none with placebo, with improvements in range of motion and function; mixed or cholestatic liver injury in a minority of patients led to a risk evaluation and mitigation strategy at FDA approval in August 2019. Published in the Lancet in 2019, it was the first randomised trial to show benefit of a drug in this disease and set the template for vimseltinib (MOTION) and emactuzumab.
Numbers are from the trial as recorded here; see the source links in the table below. This is orientation, not medical advice: ask your team how closely the trial population matches you.
Shares Sarcomas (soft tissue, bone, GIST), Small-molecule kinase inhibitors and the tag subtype-page.
Shares Tenosynovial giant cell tumour (TGCT), Sarcomas (soft tissue, bone, GIST), Small-molecule kinase inhibitors and the tag subtype-page.
Shares Tenosynovial giant cell tumour (TGCT), Sarcomas (soft tissue, bone, GIST) and the tag subtype-page.
Shares Sarcomas (soft tissue, bone, GIST) and the tag subtype-page.
Shares Sarcomas (soft tissue, bone, GIST) and the tag subtype-page.
Shares Sarcomas (soft tissue, bone, GIST) and the tag subtype-page.
Shares Sarcomas (soft tissue, bone, GIST) and the tag subtype-page.
Shares Sarcomas (soft tissue, bone, GIST) and the tag subtype-page.