Vimseltinib Approved for Symptomatic Tenosynovial Giant Cell Tumor

On February 14, 2024, the US Food and Drug Administration (FDA) approved vimseltinib for patients with symptomatic tenosynovial giant cell tumor for which surgical resection could cause worsening functional limitation or severe mobility. This regulatory decision was based on results from the MOTION trial.

In this double-blinded, multicenter trial, 123 patients in this population were randomized on a 2-to-1 basis to receive either 30 mg of vimseltinib (n = 83) twice weekly for 24 weeks or placebo (n = 40). During the following open-label period, patients could continue vimseltinib or, if randomized to receive placebo, crossover to vimseltinib treatment. Randomization was stratified by tumor location and region. The major efficacy outcome of this trial was overall response rate as assessed by blinded independent radiological review at week 25.

The objective response rate was 40% in the vimseltinib arm vs 0% in the placebo arm (P < .0001). The median duration of response was not reached. Based on an additional 6 months of follow-up, there were 28 responders with a duration of response ≥ 6 months, and 19 responders with a duration or response ≥ 9 months. In addition to meeting the primary end point of objective response rate, there were also statistically significant improvements of active range of motion, patient-reported physical functioning, and patient-reported pain in the vimseltinib arm at week 25.

The most common adverse events occurring in ≥ 20% of patients and including laboratory abnormalities were increased aspartate aminotransferase, periorbital edema, fatigue, rash, increased cholesterol, peripheral edema, face edema, decreased neutrophils, decreased leukocytes, pruritus, and increase alanine aminotransferase.

The recommended dose for vimseltinib is 30 mg twice weekly with at least 72 hours between each dose.

Source:

FDA approves vimseltinib for symptomatic tenosynovial giant cell tumor. Accessed February 14, 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-vimseltinib-symptomatic-tenosynovial-giant-cell-tumor