Dr Tap on the FDA Approval of Vimseltinib for Symptomatic TGCT

“People live with [TGCT] for long periods of time and can experience tremendous symptoms and a lot of disability from it, so to have medical options…is critical.”

William D. Tap, MD, chief of the Sarcoma Medical Oncology Service at Memorial Sloan Kettering Cancer Center, discusses the significance of the FDA approval of vimseltinib (Romvimza) for patients with symptomatic tenosynovial giant cell tumor (TGCT).

On February 14, 2025, the FDA approved vimseltinib for the treatment of patients with symptomatic TGCT for whom surgical resection will potentially cause worsening functional limitation or severe morbidity. This regulatory decision was supported by findings from the phase 3 MOTION trial (NCT05059262), in which patients in the intention-to-treat population who received vimseltinib achieved an overall response rate of 40% (95% CI, 29%-51%) at week 25 compared with 0% (95% CI, 0%-9%) in those who received placebo (difference, 40%; 95% CI, 29%-51%; P < .0001). Notably, the median duration of response was not reached among patients who received vimseltinib.

The FDA approval of vimseltinib represents a significant advancement in the management of this disease, Tap begins. Although TGCT is not typically life threatening, it can severely affect patients’ quality of life, causing chronic symptoms and substantial disability over an extended period, he explains.

Historically, surgical intervention has been the primary treatment for patients with TGCT, he says. However, patients with diffuse disease are at a high risk of recurrence and must often undergo repeat surgical procedures, which may not always be feasible or effective in the long term, he emphasizes.

Pexidartinib (Turalio) is another FDA-approved systemic therapy for the management of TGCT, Tap says, adding that its introduction to the treatment paradigm transformed patient outcomes by providing a nonsurgical treatment alternative. The approval of vimseltinib now offers a second systemic therapy option to patients, allowing for more tailored treatment approaches based on patient-specific factors, he concludes.