On February 14, 2025, the Food and Drug Administration 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.
Full prescribing information for Romvimza will be posted on Drugs@FDA.
Efficacy was evaluated in MOTION (NCT05059262), a double-blind, multicenter, randomized (2:1), placebo-controlled trial in patients with TGCT for whom surgical resection may cause worsening functional limitation or severe morbidity. Eligible patients had a confirmed diagnosis of TGCT with measurable disease (RECIST v1.1) 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 placebos could crossover to vimseltinib. Randomization was stratified by tumor location (lower limb versus all other) and region (United States versus 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% 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) was not 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 compared to the placebo arm at week 25.
The most common adverse reactions (≥20%), including laboratory abnormalities, were increased aspartate aminotransferase, periorbital edema, fatigue, rash, increased cholesterol, peripheral edema, face edema, decreased neutrophils, decreased leukocytes, pruritus, and increased alanine aminotransferase.
The recommended vimseltinib dose is 30 mg orally twice weekly, with a minimum of 72 hours between doses.
This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment.
This application was granted priority review. FDA expedited programs are described in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.
For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitateat240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.
Follow the Oncology Center of Excellence on X: @FDAOncology.
