FDA Grants Priority Review to Vimseltinib Application in Tenosynovial Giant Cell Tumor

• The FDA has granted priority review to the new drug application (NDA) of vimseltinib (DCC-3014) for the treatment of tenosynovial giant cell tumor (TGCT).
• A Prescription Drug User Fee Act (PDUFA) target action date of February 17, 2025, has been set.
• The application is supported by data from the phase 3 MOTION study (NCT05059262).

The NDA of vimseltinib, a colony stimulating factor 1 receptor (CSF1R), in TGCT has been granted FDA priority review, and a PDUFA target action date of February 17, 2025, has been set.¹

“Building upon positive results from the MOTION pivotal phase 3 study and following our recent announcement that [European Medicines Agency (EMA)] review of the vimseltinib [marketing authorization application (MAA)] 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,” said Steve Hoerter, president and chief executive officer of Deciphera Pharmaceuticals, in a press release.

The NDA is supported by data from the phase 3 MOTION study. Efficacy, safety, and patient-reported outcomes were presented at the 2024 American Society of Clinical Oncology (ASCO) Annual Meeting.

Patients were randomized 2:1 to receive 30 mg of vimseltinib twice weekly or placebo for 25 weeks. The overall response rate (ORR) at week 25 by RECIST v1.1 was 40% (95% CI, 29%-51%; P <.0001) with vimseltinib vs 0% with placebo.² This included 4 (5%) complete responses, 29 (35%) partial responses, and 42 (51%) cases of stable disease. The median duration of response (DOR) with vimseltinib was not reached (range, 0.03-11.7+). ORR by tumor volume score (TVS), a TGCT-specific MRI scoring system, was 67% (95% CI, 56%-77%; P <.0001) with vimseltinib vs 0% with placebo.

Vimseltinib also led to statistically significant and clinically meaningful improvements vs placebo for active range of motion, physical function, stiffness, and pain. Regardless of tumor response, approximately 40% of patients in the vimseltinib arm achieved a response in 3 or more clinical outcomes vs only 6% receiving placebo.

Regarding safety, most treatment-emergent adverse events (TEAEs) were grade 1 or 2, and the most common any-grade TEAEs with vimseltinib included periorbital edema (45%), fatigue (33%), face edema (31%), pruritus (29%), headache (28%), asthenia (27%), nausea (25%), increased creatinine (24%), and increased aspartate aminotransferase (23%). Serum enzyme elevations were consistent with the known mechanisms of CSF1R inhibitors, and there were no observations of cholestatic hepatotoxicity, drug-induced liver injury, or hypopigmentation of the hair or skin.

REFERENCES:

1. U.S. Food and Drug Administration accepts for priority review Deciphera’s new drug application of vimseltinib for the treatment of patients with tenosynovial giant cell tumor (TGCT). News release. Ono Pharmaceutical, Co., Ltd. August 16, 2024. Accessed August 16, 2024. https://tinyurl.com/35u4zn2m

2. Tap W, Bhadri V, Stacchiotti 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. J Clin Oncol. 2024;42(suppl 16):abstr 11500. doi:10.1200/JCO.2024.42.16_suppl.11500)