FDA Grants Full Approval for Symptomatic Tenosynovial Giant Cell Tumor Treatment

Ono Pharmaceutical, of Osaka, Japan, has announced FDA’s approval of Romvimza (vimseltinib) for adult patients with symptomatic tenosynovial giant cell tumor (TGCT), for whom surgical resection would cause potential worsening functional limitation or severe morbidity (1). Offered in capsule form, Ono’s version of vimseltinib, which is a kinase inhibitor, was developed by the company’s wholly owned subsidiary Deciphera Pharmaceuticals, of Waltham, Mass. in the United States.

The medication previously garnered fast track designation and priority review from FDA, according to an Ono Pharmaceutical press release issued Feb. 14, 2025 (1). Within the European Union, the European Medicines Agency in January 2023 had granted priority medicines (PRIME) designation to AmMax Bio’s candidate for treatment of TGCT, AMB-05X, based on Phase II trial data presented the previous fall (2).

TGCT, a rare, non-malignant type of tumor, forms within or near joints, according to Ono (1). It is caused by the dysregulation and subsequent overproduction of the CSF1 gene and, if recurring or left untreated, can damage or degenerate the affected joint and surrounding tissues, leading to the potential of significant disability. FDA based its approval on a Phase III trial (MOTION) that showed statistically significant and clinically meaningful results in patients treated with vimseltinib versus placebo after 25 weeks.

“The approval of Romvimza provides a new, much-needed, well-tolerated, and effective treatment option for people suffering from TGCT,” said Hans Gelderblom, MD, PhD, chair of the Department of Medical Oncology at Leiden University Medical Center, in the Ono press release (1). “TGCT adversely affects the lives of patients, causing significant pain, limited mobility, and stiffness. The MOTION Phase III study demonstrated Romvimza’s ability to shrink tumors along with being the first well-tolerated agent to demonstrate significant improvement in a number of other important quality-of-life measures without any observed liver injury as seen with other approved TGCT treatment. Romvimza is a differentiated treatment that has the potential to address the significant unmet needs of the TGCT community.”

“The FDA approval of Romvimza for TGCT is a crucial advancement for the TGCT community, and we believe Romvimza has the potential to become the new standard of care for people with TGCT for which surgical resection will potentially cause worsening functional limitation or severe morbidity,” said Ryota Udagawa, president and CEO of Deciphera Pharmaceuticals, in the release. “This is also an important milestone for our organization, as it is the second approved therapy discovered using Deciphera’s proprietary switch-control kinase inhibitor platform.”

While Deciphera Pharmaceuticals is, as stated above, an Ono Pharmaceutical subsidiary, another Ono partnership was announced in August 2023—a drug discovery collaboration agreement with Twist Bioscience to discover and develop innovative antibodies for autoimmune diseases utilizing Twist’s Biopharma Solutions Library of Libraries (3).

In the press release, Ono said that Deciphera Pharmaceuticals plans to make Romvimza commercially available in the US the week of February 17. A review by the European Medicines Agency (EMA) is currently underway, after a marketing authorization application was accepted by EMA in July 2024 (1).

References

1. Ono Pharmaceutical. U.S. FDA Grants Full Approval of Deciphera’s Romvimza (vimseltinib) for the Treatment of Symptomatic Tenosynovial Giant Cell Tumor (TGCT). Press Release. Feb. 14, 2025.
2. AmMax Bio. AmMax Bio’s AMB-05X Receives EMA PRIME Designation for the Treatment of Tenosynovial Giant Cell Tumor (TGCT). Press Release. Jan. 19, 2023.
3. Ono Pharmaceutical. Ono Enters into a Drug Discovery Collaboration Agreement with Twist Bioscience to Discover Novel Antibodies for Autoimmune Diseases. Press Release. Aug. 31, 2023.