WALTHAM, Mass– Deciphera Pharmaceuticals and Ono Pharmaceutical Co., Ltd. have announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending approval of vimseltinib (brand name ROMVIMZA™) for the treatment of tenosynovial giant cell tumor (TGCT), a rare and debilitating joint condition.
If approved, vimseltinib will become the first therapy in the European Union (EU) with regulatory backing for TGCT in adult patients experiencing significant physical dysfunction for whom surgery is not viable or poses unacceptable risks.
The CHMP recommendation represents a major regulatory milestone and paves the way for a final decision from the European Commission, which is expected in the second quarter of the fiscal year ending March 31, 2026.
“CHMP’s positive opinion is an important milestone for the TGCT community in the European Union, where there are currently no approved treatments for TGCT,” said Ryota Udagawa, President and CEO of Deciphera. “We look forward to building upon this momentum to bring vimseltinib to patients globally who are in urgent need of new treatment options.”
The recommendation is based on strong clinical data from Deciphera’s pivotal Phase 3 MOTION trial, which evaluated vimseltinib in patients with symptomatic TGCT not amenable to surgery and with no prior anti-CSF1/CSF1R therapy. Patients who had received imatinib or nilotinib were allowed.
According to the company, vimseltinib demonstrated a statistically significant and clinically meaningful objective response rate (ORR) at Week 25, with 40% of patients in the treatment group responding to therapy, compared to none in the placebo group (p < 0.0001), as assessed by blinded independent radiologic review using RECIST v1.1 criteria.
Patients receiving vimseltinib also showed notable improvements in joint mobility, physical functioning, and pain levels, further reinforcing the drug’s therapeutic potential. The safety profile was deemed manageable and consistent with earlier Phase 1/2 findings.
Vimseltinib, a selective CSF1R inhibitor, represents a novel approach to treating TGCT, which can severely impact quality of life through joint pain, swelling, and reduced mobility. Surgical removal is often difficult or ineffective due to tumor location and recurrence.
With the CHMP’s endorsement, Deciphera is now one step closer to offering the first non-surgical treatment option for TGCT patients in Europe.
