Ono Pharmaceutical, one of the largest pharmaceutical companies in Japan headquartered in Chuo-ku, Osaka, has received approval from the US Food and Drug Administration for its kinase inhibitor Romvimza (vimseltinib) to treat tenosynovial giant cell tumour (TGCT).
Tenosynovial giant cell tumour 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.
Ono Pharmaceutical obtained Romvimza as part of its purchase of Deciphera Pharma. The approval follows positive phase III MOTION study results (NCT05059262), which showed a 40 per cent overall response rate at 25 weeks, assessed by a blinded independent radiological review, compared with zero per cent in a matched placebo group. Among treated patients, 85 per cent maintained a response for at least 6-month, while over half saw benefits for more than 9-month. The reduced tumour size correlated with improved range of motion, physical functioning, and pain reduction.
Vimseltinib has been approved to treat adults with symptomatic tenosynovial giant cell tumour – a rare non-cancerous tumour that causes inflammation and destruction of joints – if there is a concern that surgery will lead to a serious loss of function or other complications.
Ono Pharmaceutical says US Food and Drug Administration approval for Romvimza as a treatment for tenosynovial giant cell tumour is setting up a market tussle with Daiichi Sankyo. It becomes the second US Food and Drug Administration-approved drug for the disease after Daiichi Sankyo's Turalio (pexidartinib), a CSF1R, KIT, and FLT3 inhibitor that was cleared by the US Food and Drug Administration in 2019 for tenosynovial giant cell tumour associated with severe morbidity or functional limitations and not responsive to improvement with surgery.
In the majority of patients receiving Romvimza, the duration of response was at least six months, while more than half saw a benefit for more than nine months, and the reduced tumour size was accompanied by improvements in active range of motion, patient-reported physical functioning, and patient-reported pain.
According to study investigator Hans Gelderblom of Leiden University Medical Centre in the Netherlands, the new drug is ""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 tenosynovial giant cell tumour treatment."