Brussels, Belgium – September 17, 2025 – In a landmark development for rare disease patients, the European Commission (EC) has approved ROMVIMZA™ (vimseltinib), developed by Deciphera Pharmaceuticals and Ono Pharmaceutical Co., Ltd., as the first and only therapy for tenosynovial giant cell tumor (TGCT) in the European Union. This approval marks a crucial step forward in addressing an unmet medical need for patients where surgical options are no longer viable or carry unacceptable risks.
Science Significance
TGCT is a rare, locally aggressive tumor driven by overexpression of the CSF1 gene, which recruits inflammatory cells into the lesion. The disease can cause severe pain, stiffness, swelling, and joint damage, leading to disability. Surgery has long been the only option, but recurrence is common, particularly in diffuse-type TGCT.
ROMVIMZA™ (vimseltinib) is a switch-control kinase inhibitor targeting the CSF1 receptor pathway. Its approval is based on robust data from the Phase 3 MOTION trial and a Phase 1/2 study, which demonstrated statistically significant and clinically meaningful improvements in patient mobility, pain reduction, and quality of life compared to placebo.
Notably, at Week 97 of MOTION, 23% of patients achieved a complete response, highlighting vimseltinib’s potential as a disease-modifying therapy for TGCT.
Regulatory Significance
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The EC’s decision grants Deciphera and Ono the ability to commercialize ROMVIMZA™ across all 27 EU member states. This follows earlier approval in the United States, positioning vimseltinib as a global standard of care for TGCT patients with no surgical options.
This is the first approved systemic therapy for TGCT in the EU, setting a precedent for rare oncology drug approvals and validating Deciphera’s kinase inhibitor platform. Regulatory agencies worldwide are expected to look closely at Europe’s decision as a reference point for further submissions.
Business Significance
For Deciphera and Ono, the approval represents a significant commercial and strategic milestone. Deciphera, now a subsidiary of Ono, will leverage Ono’s global infrastructure to accelerate patient access. The companies are well-positioned to expand into additional markets, reinforcing their oncology leadership.
The approval also strengthens Deciphera’s portfolio, which already includes QINLOCK® (ripretinib) for gastrointestinal stromal tumor (GIST). Together, these therapies illustrate the company’s ability to advance first- or best-in-class kinase inhibitors into the global market.
This achievement also enhances Ono’s international footprint, aligning with its strategy of expanding beyond Japan into Europe and the U.S. with high-impact therapies.
Patients’ Significance
For patients, the approval of ROMVIMZA™ provides the first-ever systemic treatment option for TGCT in Europe. Until now, the only recourse was repeated surgeries, often leading to joint degeneration, worsening mobility, and disability.
Patient advocates and clinicians alike welcomed the news, emphasizing that ROMVIMZA™ addresses not only tumor burden but also restores quality of life by improving joint function, reducing stiffness, and alleviating pain. For those with advanced or diffuse TGCT, this drug offers hope where none previously existed.
Policy Significance
The approval reflects the EU’s continued commitment to advancing therapies for rare diseases and cancers with high unmet needs. By authorizing ROMVIMZA™, the European Commission underscores the importance of expedited regulatory pathways for conditions that lack effective treatment options.
This decision may also prompt national health authorities across Europe to consider accelerated reimbursement frameworks for rare oncology drugs, ensuring rapid patient access while balancing cost-effectiveness in healthcare systems.
Transaction Highlights
The European Commission has approved ROMVIMZA™ (vimseltinib) in the European Union, making it the first and only therapy authorized for the treatment of tenosynovial giant cell tumor (TGCT) in adults whose disease causes clinically relevant physical function deterioration and for whom surgery is not a viable option. The approval was based on compelling results from the pivotal Phase 3 MOTION trial and a supporting Phase 1/2 study, which together demonstrated statistically significant and clinically meaningful improvements in joint mobility, physical function, pain reduction, and overall quality of life compared to placebo. In addition, the MOTION trial showed durable responses, with 23% of patients achieving complete responses at Week 97, and the safety profile of vimseltinib was consistent with prior studies and considered manageable. With this milestone, Deciphera and Ono will leverage their global commercial infrastructure to ensure rapid access for patients across the EU, delivering the first systemic therapy option for TGCT and transforming care for individuals facing disability due to this rare disease.
Source: Deciphera Pharmaceuticals, Inc. Press Release
