Vimseltinib

An Ono Pharma drug for a type of tumor affecting tissues of the joints is now FDA approved, giving the Japanese company a product with dosing and safety advantages over the Daiichi Sankyo product that was the first systemic therapy for this rare condition. The approval of the Ono drug, vimseltinib, covers the treatment of adults whose tenosynovial giant cell tumor (TGCT) cannot be surgically removed. The twice-weekly capsule will be marketed under the brand name Romvimza. TGCT stems from a genetic abnormality that results in overexpression of a protein called colony-stimulating factor receptor. This protein recruits other tumor-promoting cells, leading to the growth of tumors in and around joints. While these tumors are not malignant, they damage surrounding tissues and cause pain and swelling. These tumors also limit movement of the joint. Surgery is the standard treatment, but these tumors can come back and for some patients, surgery can lead to more complications. presented by Artificial Intelligence What Keeps Healthcare CIOs Up at Night: Balancing Technology Investments with Consumer Expectations When it comes to managing inbound phone calls, underperformance has devastating cost implications. By Stephanie Baum Romvimza inhibits CSF-1. The small molecule was initially developed by Waltham, Massachusetts-based Deciphera Pharmaceuticals, whose drugs are based on a technology that targets a region of an enzyme that activates it or inactivates it like an on-off switch. Last year, Ono acquired Deciphera in a $2.4 billion deal. In addition to Romvimza, Ono gained Qinlock, a cancer drug that Deciphera steered to a 2020 FDA approval in gastrointestinal stromal tumor. Inhibiting CSF-1 to treat TGCT was validated by Turalio, a twice-daily Daiichi Sankyo drug that won its FDA approval in this indication in 2019. But Turalio’s label carries a black box warning for the risk of liver toxicity. This complication was observed in some patients treated with the drug in clinical trials; there was one fatality. Daiichi Sankyo must provide a Risk Evaluation and Mitigation Strategy, a plan that informs clinicians and patients about the safety risks. In 2020, European regulators refused marketing authorization for Turalio. Clinical tests of Ono’s Romvimza showed no reports of liver toxicity. But due to the serious liver injury observed in Daichii’s CSF-1 inhibitor, Romvimza’s label advises avoiding use of the drug by patients who already have high levels of liver enzymes in the blood. The FDA also recommends liver tests before and during treatment with Romvinmza. In its 123-patient Phase 3 study, twice-weekly Romvimza led to a 40% overall response rate measured at week 25 compared to 0% in the placebo arm. Results also showed statistically significant improvement in active range of motion as well as patient reported measures of physical functioning and pain. Romvimza is still under regulatory review in Europe. Sponsored Post Tips for Greater Healthcare Practice Success in 2025 Join us on February 26 at 3 pm ET, and we’ll dive into the key areas where practices are losing time and money and provide solutions to overcome them. By Elation Health and MedCity News “The MOTION Phase 3 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,” Dr. Hans Gelderblom, chair of the department of medical oncology at Leiden University Medical Center, said in Ono’s approval announcement. “Romvimza is a differentiated treatment that has the potential to address the significant unmet needs of the TGCT community.” There are other companies vying to treat TGCT. Merck KGaA could compete with pimicotinib, a CSF-1 inhibiting small molecule being developed under a commercialization agreement with Abbisko Therapeutics. Last November, the two companies announced this drug met the main goal of its pivotal study. Meanwhile, SynOx Therapeutics has reached Phase 3 testing with emactuzumab, an antibody designed to block the CSF-1 receptor. Last year, the privately held company raised $75 million in Series B financing for the pivotal test of this molecule.

TGCT is a rare, non-cancerous tumour affecting the joints, tendons, or joint tissue. Credit: Kinga Krzeminska via Getty Images. 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. 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. GloblData is the parent company of Pharmaceutical Technology. 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.