Ono picked up Romvimza—previously known by its active ingredient vimseltinib—from its $2.4 billion acquisition of Deciphera Pharmaceuticals in April 2024.
Ono Pharmaceuticals on Friday won the FDA’s greenlight for vimseltinib for the treatment of tenosynovial giant cell tumor. The drug will be marketed under the brand name Romvimza.
With its regulatory win, Ono will now go toe-to-toe with fellow Japanese pharma Daiichi Sankyo in the tenosynovial giant cell tumor (TGCT) market. Daiichi owns Turalio, which is indicated for TGCT in adult patients with severe morbidity or functional limitations and not amenable to improvement with surgery. In its latest quarterly report—covering the third quarter of its 2024 fiscal year—Daiichi reported a nearly 25% increase in Turalio sales to ¥5.1 billion (approximately $33.6 million)
Like Turalio, Romvimza is an oral drug that can be used to treat TGCT patients for whom surgery “will potentially cause worsening of functional limitation or severe morbidity,” according to its label.
To overcome Turalio’s first-to-market advantage, Ono is offering a more convenient treatment schedule—unlike Daiichi’s drug, which needs to be taken twice-daily, Romvimza can be taken twice-weekly, with at least 72 hours between doses.
Turalio also carries a boxed warning for hepatotoxicity, warning patients and prescribers of its risk to cause “serious and potentially fatal liver injury.” Romvimza has no such warning attached.
TGCT refers to a group of rare tumors that, while not typically life-threatening, can severely affect the joints and surrounding tissues, leading to significant disability. If left unchecked, TGCT can necessitate amputation.
Romvimza is a kinase inhibitor that works by blocking the colony-stimulating factor 1 (CSF1) receptor and preventing the proliferation of cells carrying the protein. In TGCT, this mechanism of action could potentially prevent the tumor mass from spreading.
Ono obtained rights to Romvimza in April 2024, when it dropped $2.4 billion to acquire the Boston biotech Deciphera Pharmaceuticals.
Romvimza’s approval on Friday was supported by data from the Phase III MOTION study, which enrolled TGCT patients who had not been previously exposed to anti-CSF1/CSF1R therapy. Results showed that Romvimza elicited a 40% objective response rate at 25 weeks, versus 0% in placebo comparators. Ono’s drug also resulted in “statistically significant and clinically meaningful improvements” in physical function, range of motion and pain.
Ono expects Romvimza to be available in the U.S. in the coming days.
