Ono Pharmaceutical has secured approval from the US Food and Drug Administration (FDA) for its kinase inhibitor Romvimza (vimseltinib) to treat tenosynovial giant cell tumour (TGCT), introducing competition to Daiichi Sankyo’s Turalio (pexidartinib).
TGCT is a rare, non-cancerous tumour affecting the joints, tendons, or joint tissue. It is linked to a CSF1 gene mutation that causes abnormal cell growth, joint pain, stiffness, and inflammation. While surgical removal is often used as the first-line treatment, recurrence is common. CSF1 inhibitor Romvimza has been approved for patients where surgery could cause severe loss of function or other complications.
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Romvimza’s approval positions it as a competitor to Daiichi Sankyo’s Turalio, which became the first FDA-approved drug for TGCT in August 2019. Daiichi’s drug is not only a CSF1 inhibitor, but also a blocker of KIT and FLT3. Turalio is taken orally twice daily, whereas Ono’s therapy is taken orally twice weekly. The differences in dosing schedule could provide a competitive advantage for Ono as fewer doses are generally preferred by patients and lead to higher adherence rates.
Additionally, Daiichi’s Turalio comes with a black box warning for severe and potentially fatal liver toxicity. Due to this risk, it is only available through a risk evaluation and mitigation strategy (REMS) programme. In contrast, Romvimza does not require a REMS programme, though patients will need liver enzyme monitoring. Blood tests are required before starting Ono’s treatment, twice a month for the first two months, and then once every three months for the first year.
Turalio generated Y5.3bn ($35m) in 2023, up from Y3.8bn ($25m) in 2022. According to GlobalData’s Pharma Intelligence Center, Turalio is forecast to generate $54m in global sales by 2030 while Romvimza is projected to generate $152m.
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Ono inherited Romvimza as part of its $2.4bn purchase of Deciphera Pharma in April 2024. The approval follows positive Phase III MOTION study results (NCT05059262), which showed a 40% overall response rate (ORR) at 25 weeks, assessed by a blinded independent radiological review, compared with 0% in a matched placebo group. Among treated patients, 85% maintained a response for at least six months, while over half saw benefits for more than nine months. The reduced tumour size correlated with improved range of motion, physical functioning, and pain reduction.
Elsewhere in the TGCT space, another big pharma company is edging towards bringing a new drug to market. In November 2024, MSD reported positive results with its once-daily oral CSF-1R inhibitor pimicotinib, licensed from Chinese biotech Abbisko Therapeutics. In the Phase III MANEUVER study (NCT05804045), pimicotinib achieved a 54% ORR at 25 weeks, compared to 3.2% for placebo, meeting its primary endpoint.
