FDA Accepts NDA and Priority Review Application for Vimseltinib in Tenosynovial Giant Cell Tumors

The FDA accepted the new drug application for vimseltinib (Deciphera Pharmaceuticals) for priority review. The drug is a colony stimulating factor 1 receptor for patients with tenosynovial giant cell tumor (TGCT). Vimseltinib has a Prescription Drug User Fee Act goal date of February 17, 2025.¹

Image Credit: Sebastian Kaulitzki - stock.adobe.com

“Building upon positive results from the MOTION pivotal phase 3 study and following our recent announcement that [European Medicine Agency] review of the vimseltinib [Marketing Authorization Application] has begun, we are excited to initiate the regulatory review process in the US and we look forward to working with the FDA to deliver a new treatment option to patients with TGCT,” Steve Hoerter, president and CEO of Deciphera Pharmaceuticals, said in a press release.¹

MOTION (NCT05059262) is a global, phase 3, double-blind study of vimseltinib in patients with symptomatic TGCT that are unable to have surgery. Investigators randomized treatment 2:1 with vimseltinib 30 mg twice weekly or the matching placebo for 24 weeks, according to the study authors. The primary end point was objective response rate (ORR) at week 25, with secondary end points including ORRR at week 25 per tumor volume score, change from baseline in active range of motion (ROM) of the affected joint and patient-reported outcomes, wort stiffness numeric rating scale, EuroQol Visual Analog Scale, and brief pain inventory worst pain response. The investigators also evaluated the safety of vimseltinib.²

The investigators assigned 123 patients to treatment (83 to vimseltinib, 40 to the placebo) between January 21, 2022, and February 21, 2023. The median age was 44 years, 59% were female, and most of the primary disease location was the knee at 67%, according to the authors. Approximately 11% of patients on vimseltinib and 13% on the placebo discontinued treatment before week 25 and 1 individual in the placebo group did not receive any study drug.^2,3

The ORR was 40% for vimseltinib and 0% in the placebo group. The study authors also said there were significant improvements from baseline to 25 weeks in active ROM at 18.4% and 3.8%, respectively, physical function with a 3.3-point difference between the 2 arms, worst stiffness with a –1.8-point difference, and health status with a 7.4-point difference.^2,3

Further, there were significantly more pain responders with vimseltinib (48%) compared with the placebo (23%). Most treatment-emergence adverse events (TEAEs) were grade 1 or 2, with the only grade 3 or 4 occurring in 5% or more of individuals treated with vimseltinib being blood creatine phosphokinase, according to the study authors. Data did not show support of cholestatic hepatoxicity or drug-induced liver injury. There was 1 patient who experienced a serious TEAE of subcutaneous abscess when treated with vimseltinib.^2,3

The results were presented at the 2024 American Society of Clinical Oncology Annual meeting and published in Lancet.¹

REFERENCES

1. U.S. Food and Drug Administration Accepts for Priority Review Deciphera’s New Drug Application for Vimseltinib for the Treatment of Patients with Tenosynovial Giant Cell Tumor (TGCT). News release. Ono Pharma. August 15, 2024. Accessed August 19, 2024. https://www.businesswire.com/news/home/20240815607407/en/U.S.-Food-and-Drug-Administration-Accepts-for-Priority-Review-Deciphera%E2%80%99s-New-Drug-Application-for-Vimseltinib-for-the-Treatment-of-Patients-with-Tenosynovial-Giant-Cell-Tumor-TGCT

2. Tap WD, Bhadri V, Stacchiotti S, Bauer S, et al. Efficacy, safety, and patient-reported outcomes of vimseltinib in patients with tenosynovial giant cell tumor: Results from the phase 3 MOTION trial. Journal of Clinical Oncology. doi:https://doi.org/10.1200/JCO.2024.42.16_suppl.11500

3. Gelderblom H, Bhadri V, Stacchiotti S, et al. Vimseltinib versus placebo for tenosynovial giant cell tumour (MOTION): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2024;403(10445):2709-2719. doi:10.1016/S0140-6736(24)00885-7