The MHRA has approved vimseltinib (Romvimza) for adults with symptomatic tenosynovial giant cell tumour when surgery is unsuitable.
Written By: Kalyani Boharapi,
M.Pharm (Reg. Affairs)
Reviewed By: Pharmacally Editorial Team
The UK Medicines and Healthcare products Regulatory Agency (MHRA) has approved vimseltinib (Romvimza) for adults with symptomatic tenosynovial giant cell tumour (TGCT) when the disease affects movement or surgery is not an appropriate option. The 14 August 2026 approval gives patients with this rare, locally aggressive tumour access to an oral CSF1R inhibitor and follows regulatory approvals in the US and European Union.
Targeted treatment for a rare joint tumour
TGCT is a rare, non-cancerous tumour that develops around joints and tendons, most often affecting the knee or ankle. Although it does not typically metastasise, the disease can cause persistent pain, swelling, stiffness and restricted movement. In patients with diffuse or symptomatic disease, repeated surgery can lead to functional impairment, while some tumours cannot be removed safely.
Vimseltinib is an oral colony-stimulating factor 1 receptor (CSF1R) inhibitor. TGCT cells produce excessive CSF1, which activates CSF1R and promotes recruitment and proliferation of cells that contribute to tumour growth. By inhibiting CSF1R signalling, vimseltinib reduces this tumour-supporting cellular activity.
MOTION trial established the clinical benefit
The pivotal MOTION trial (NCT05059262) was a double-blind, multicentre, randomised, placebo-controlled study involving 123 adults with TGCT for whom surgical resection could cause worsening functional limitation or severe morbidity. Patients were randomised 2:1 to vimseltinib or placebo, with 83 receiving vimseltinib and 40 receiving placebo during the 24-week double-blind period.
At week 25, the overall response rate (ORR) assessed by blinded independent radiological review was 40% with vimseltinib versus 0% with placebo (95% CI, 29%-51% vs 0%-9%; P<0.0001). The response comprised complete responses in 5% and partial responses in 35% of patients receiving vimseltinib.
The tumour response was accompanied by functional benefits. Vimseltinib produced statistically significant improvements in active range of motion, patient-reported physical functioning and patient-reported pain compared with placebo. Among responders, the median duration of response had not been reached at the analysis cutoff; 85% of responders maintained a response for at least six months and 58% for at least nine months.
Twice-weekly dosing with at least 72 hours between doses
Romvimza is administered as an oral capsule twice weekly, with at least 72 hours between doses, and can be taken with or without food. The MHRA has placed vimseltinib under additional monitoring and advises against its use during pregnancy.
Common adverse reactions include increased liver enzymes, periorbital and peripheral oedema, fatigue, rash, increased cholesterol, facial oedema, decreased neutrophil and leukocyte counts, and pruritus. The FDA also identifies liver enzyme abnormalities as an important safety consideration.
MHRA highlights continued safety surveillance
Julian Beach, MHRA Executive Director for Healthcare Quality and Access, said:
“The approval of vimseltinib provides a new treatment option for patients with TGCT who have symptoms but are unsuitable for surgery.”
He added that the agency would continue to keep the medicine’s safety under close review.
The MHRA granted the marketing authorisation to Deciphera Pharmaceuticals through the International Recognition Procedure Route B on 14 August 2026.
UK approval follows US and EU authorisations
Vimseltinib received US FDA approval on 14 February 2025 for adults with symptomatic TGCT for whom surgical resection could cause worsening functional limitation or severe morbidity. The European Commission subsequently granted EU-wide marketing authorisation on 17 September 2025 for adults with symptomatic TGCT associated with clinically relevant deterioration in physical function when surgical options have been exhausted or would cause unacceptable morbidity or disability.
The UK approval therefore extends availability of a targeted systemic treatment across three major regulatory markets. Continued post-authorisation surveillance will be important because long-term safety data remain more limited than the evidence supporting short-term tumour response and functional improvement.
Reference
Vimseltinib (romvimza) approved for use in adults to treat tenosynovial giant cell tumours – GOV.UK
About the Writer
Kalyani Boharapi (LinkedIn) is a pharmacy professional and healthcare writer currently pursuing an M.Pharm in Regulatory Affairs at Dr. D. Y. Patil College of Pharmacy, with interests in pharmaceutical regulations, drug development, and healthcare innovation. She has academic exposure to dossier preparation, scientific writing, and regulatory documentation. Kalyani has also completed certification courses in Generative AI, AI in Pharma, and Bioinformatics, and actively participates in pharmaceutical conferences to stay updated with emerging trends and advancements in the healthcare and pharmaceutical industry.