Originally published by our sister publication Clinical Oncology News
By Clinical Oncology News Staff
The FDA has approved vimseltinib (Romvimza, Deciphera Pharmaceuticals), 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.
Efficacy was evaluated in MOTION (ClinicalTrials.gov Identifier: 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 version 1.1) 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 placebo could cross over to vimseltinib. Randomization was stratified by tumor location (lower limb vs. all others) and region (United States vs. non-U.S.). 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<0.0001). Median duration of response (DOR) was not reached in the vimseltinib arm, and based on an additional six months of follow-up, 28 responders (85%) had a DOR for at least six months and 19 (58%) had a DOR for at least nine months. The primary end point 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 with 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 full prescribing information for vimseltinib will be posted on Drugs@FDA.
Based on a press release from the FDA.
