[Comment] Pimicotinib: a new agent for an orphan disease

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Source: The Lancet

Original: https://www.thelancet.com/journals/lancet/article/PIIS0140-6736(26)00191-1/fullt...

Published: 2026-03-05T23:30:01Z

Tenosynovial giant cell tumor (TGCT), formerly known as pigmented villonodular synovitis, is a rare and locally aggressive connective tissue tumor that occurs mainly in young adults.[1] The tumor is caused by overexpression of colony-stimulating factor (CSF-1), which arises as a result of fusion genes or transcriptional deregulation with the macrophage receptor CSF-1R.[1] Symptoms of TGCT include joint swelling, pain, functional impairment, and potential joint destruction.[1] These symptoms have a significant negative impact on the patients' quality of life and work capacity.[1] Pimicotinib represents a new treatment approach for this rare disease.[1]