FDA Grants Priority Review to Vimseltinib Application in Tenosynovial Giant Cell Tumor
A Prescription Drug User Fee Act target action date of February 17, 2025, has been set for a decision on the vimseltinib application.
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- 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.
