This authorisation is specifically for patients who have symptomatic TGCT, with significant physical functional decline, and for whom surgical intervention is either not an option or would lead to excessive disability or morbidity.
The approval by the commission is grounded in the safety and efficacy outcomes from the pivotal Phase III MOTION trial of vimseltinib, against placebo, in TGCT subjects ineligible for surgery and who had not received anti-colony-stimulating factor 1/colony colony-stimulating factor 1 receptor (CSF1/CSF1R) therapy previously, except for treatments with imatinib or nilotinib.
The study’s positive results were also corroborated by a Phase I/II trial of the therapy.
The primary endpoint was backed by improvements in patients’ range of motion, physical functioning, and pain levels in the group receiving vimseltinib against the placebo group, at the 25-week assessment point.
Furthermore, the therapy demonstrated clinically meaningful progress across all six secondary goals, which included objective response rate, range of motion, stiffness, physical function, pain, and overall quality of life.
An analysis conducted at week 97 showed that 23% of subjects administered the therapy experienced a complete response as determined by independent radiological review, with the median duration to achieve this response being 11.5 months.
The safety profile of the therapy was found to be manageable and in line with previous Phase I/II trial findings.
According to the company, TGCT is a condition that arises due to a CSF1 gene’s “translocation” leading to the overproduction of CSF1, which in turn attracts CSF1R-positive inflammatory cells to the affected area.
Deciphera, a member of Ono Pharmaceutical, develops medicines utilising its switch-control kinase inhibitor platform.
Deciphera CEO and president Ryota Udagawa said: “The European Commission’s approval of vimseltinib for TGCT is a significant milestone for Deciphera, Ono, and TGCT patients across the European Union who are in need of a non-invasive treatment option.
“We are excited to leverage our global commercial infrastructure to bring vimseltinib to these patients.”