FDA approves vimseltinib for symptomatic tenosynovial giant cell tumor

Regulatory approval: On Feb 14, 2025, the FDA approved vimseltinib (Romvimza; Deciphera), a kinase inhibitor, for adults with symptomatic tenosynovial giant cell tumor (TGCT) when surgical resection would potentially worsen functional limitation or cause severe morbidity. Approval was supported by the randomized, placebo-controlled MOTION trial (NCT05059262), where week-25 blinded ORR was 40% with vimseltinib vs 0% with placebo (p<0.0001). Median DOR was not reached at the time of analysis.

FDA approval: vimseltinib for symptomatic TGCT (Feb 14, 2025)

Event type: Regulatory approval

On February 14, 2025, the FDA approved vimseltinib (Romvimza; Deciphera Pharmaceuticals, LLC), a kinase inhibitor, for adult patients with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity.

Key efficacy evidence (MOTION; NCT05059262)

  • Design: Double-blind, multicenter, randomized (2:1), placebo-controlled trial in TGCT where surgery may cause worsening functional limitation or severe morbidity.
  • Eligibility: Confirmed TGCT with measurable disease (RECIST v1.1); ≥1 lesion ≥2 cm.
  • Treatment (Part 1, 24 weeks): vimseltinib 30 mg twice weekly vs placebo; open-label continuation/crossover in Part 2.
  • Randomized: 123 patients (83 vimseltinib; 40 placebo).
  • Primary endpoint: ORR by blinded independent radiological review at week 25.
    • ORR 40% (95% CI, 29–51) with vimseltinib vs 0% (95% CI, 0–9) with placebo; p<0.0001.
    • Median DOR not reached; with ~6 additional months’ follow-up, 85% of responders had DOR ≥6 months and 58% had DOR ≥9 months.
  • Supportive findings: statistically significant improvements in active range of motion, patient-reported physical functioning, and pain at week 25 vs placebo.

Safety and dosing (from FDA communication)

  • Common adverse reactions (≥20%), including lab abnormalities: increased AST/ALT, periorbital/facial/peripheral edema, fatigue, rash, increased cholesterol, pruritus, decreased neutrophils/leukocytes.
  • Recommended dose: 30 mg orally twice weekly, ≥72 hours between doses.

The application received priority review; full prescribing information is expected on Drugs@FDA.

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