FDA

Romvimza OK’d for tenosynovial giant cell tumor

February 20, 2025

Brand name: Romvimza

Generic name: vimseltinib

Manufacturer: Deciphera Pharmaceuticals

Approval date: February 14, 2025

FDA approved Romvimza (vimseltinib), a kinase inhibitor, for adults with symptomatic tenosynovial giant cell tumor (TGCT) for which surgical resection will potentially cause worsening functional limitation or severe morbidity.

Efficacy

Approval was based on results from the phase 3 MOTION trial (NCT05059262) involving patients with TGCT for whom surgical resection may cause worsening functional limitation or severe morbidity. Eligible patients had a confirmed diagnosis of TGCT with at least one lesion having a minimum size of 2 cm.

Patients were randomized to placebo or vimseltinib, 30 mg twice weekly administered for 24 weeks, during the double-blind period (Part 1). During the open-label period (Part 2), patients could continue vimseltinib and those receiving placebo could crossover to vimseltinib. Randomization was stratified by tumor location (lower limb vs. all other) and region (U.S. vs. Non-US). A total of 123 patients were randomized: 83 to the vimseltinib arm and 40 to placebo during Part 1.

The major efficacy outcome measure was overall response rate (ORR) assessed by blinded independent radiological review at week 25. ORR was 40% (95% confidence interval [CI], 29%, 51%) in the vimseltinib arm and 0% (95% CI, 0%, 9%) in the placebo arm (p-value <0.0001). Median duration of response (DOR) wasn’t reached in the vimseltinib arm, and based on an additional 6 months of follow-up, 28 responders (85%) had a DOR ≥6 months and 19 (58%) had a DOR ≥9 months. The primary endpoint was supported by statistically significant improvements in active range of motion, patient-reported physical functioning, and patient-reported pain observed in the vimseltinib arm vs. the placebo arm at week 25.

Safety

The most common adverse reactions (≥20%), including lab abnormalities, were increased ALT/AST, periorbital edema, fatigue, rash, increased cholesterol, peripheral edema, face edema, decreased neutrophils, decreased leukocytes, and pruritus.

Recommended dose

The recommended dosated of vimseltinib is 30 mg PO twice weekly, with a minimum of 72 hours between doses.

Sources:

FDA approves vimseltinib for symptomatic tenosynovial giant cell tumor. Food and Drug Administration. 2025. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-vimseltinib-symptomatic-tenosynovial-giant-cell-tumor

U.S. FDA Grants Full Approval of Deciphera’s ROMVIMZA™ (vimseltinib) for the Treatment of Symptomatic Tenosynovial Giant Cell Tumor (TGCT). Deciphera Pharmaceuticals. 2025. https://www.deciphera.com/news/us-fda-grants-full-approval-decipheras-romvimzatm-vimseltinib-treatment-symptomatic