FDA Approves Romvimza for Treatment of Tenosynovial Giant Cell Tumor

The U.S. Food and Drug Administration (FDA) has approved Romvimza (vimseltinib) for the treatment of tenosynovial giant cell tumor (TGCT). The drug, developed by Ono Pharmaceuticals, is intended for patients who are not suitable candidates for surgery due to potential worsening of functional limitations or severe morbidity.
Romvimza is a kinase inhibitor that targets the colony-stimulating factor 1 (CSF1) receptor, preventing the proliferation of cells carrying the protein. This mechanism may help prevent tumor mass expansion in TGCT, which, while not typically life-threatening, can cause joint damage and disability.

The approval was supported by data from the Phase III MOTION study, which evaluated Romvimza in TGCT patients who had not previously received anti-CSF1/CSF1R therapy. Results showed a 40% objective response rate at 25 weeks in the treatment group, compared to 0% in the placebo group. The study also reported significant improvements in physical function, range of motion, and pain management.
Romvimza will compete with Daiichi Sankyo’s Turalio, another TGCT treatment. Unlike Turalio, which is taken twice daily, Romvimza requires only twice-weekly dosing with at least 72 hours between doses. Additionally, Turalio carries a boxed warning for hepatotoxicity due to its potential to cause severe liver injury, a warning not present in Romvimza’s labeling.
Ono acquired Romvimza as part of its $2.4 billion acquisition of Deciphera Pharmaceuticals in April 2024. The company expects to make the drug available in the U.S. in the coming days.

According to the U.S. Centers for Disease Control and Prevention (CDC), cases of TGCT can lead to severe joint impairment and, in extreme cases, amputation. While TGCT is rare, effective treatments are essential to improve patient outcomes.