Sarcoma UK today (Wednesday 20 August) welcomed the European Commission’s groundbreaking approval of Ogsiveo (nirogacestat) as the first authorised drug treatment for desmoid tumours, while welcoming the upcoming evaluation of the drug by UK regulators to allow access for British patients living with these devastating rare tumours.
The historic decision marks a turning point for people living with desmoid tumours in the UK, who have previously faced a complete absence of approved treatment options for their aggressive, locally invasive soft tissue growths. With only 5-6 people in every million affected by these rare tumours, patients often feel isolated and underserved by existing healthcare pathways.
‘This approval represents a beacon of hope for desmoid tumour patients who have waited far too long for an effective drug treatment,’ said Dr Sorrel Bickley, Director of Research, Policy and Support at Sarcoma UK. ‘However, hope alone is not enough. We await the NICE and the MHRA appraisals of Ogsiveo so that UK patients can access this life-changing therapy without delay.’
Desmoid tumours, while rare, can be devastating in their impact. These aggressive growths cause severe pain, disfigurement and loss of mobility, with patients often facing multiple surgeries and high recurrence rates. The psychological toll on patients and families is immense, with many feeling abandoned by a healthcare system that has had no approved treatments to offer.
The EU approval is based on compelling evidence from the phase 3 DeFi trial, which demonstrated that Ogsiveo reduced the risk of disease progression by 71% compared to placebo. Crucially for patients, the treatment also delivered early and sustained improvements in pain, physical functioning and overall quality of life – outcomes that matter profoundly to those living with these tumours.
Dr Bickley said: ‘The clinical trial results are remarkable, but what matters most is the real-world impact on patients’ lives. We’ve heard from too many families who have watched loved ones suffer without adequate treatment options. This approval changes that narrative, but only if we can secure timely access in the UK.’
The charity is particularly encouraged by the therapy’s dual benefits of tumour control and symptom relief. Professor Bernd Kasper, who led the pivotal trial, noted the treatment’s ‘meaningful antitumour activity and significant improvement in desmoid tumour symptoms, including a significant reduction in pain’.
Dr Bickley added: ‘We should move ahead swiftly with this drug. Rare tumour patients cannot afford to wait. Every month of delay means more patients suffering without access to a treatment that could potentially transform their lives.’
Sarcoma UK also highlighted the broader significance of the approval for the rare cancer community, noting that it demonstrates the importance of continued research investment and regulatory support for orphan diseases.
About desmoid tumours: Desmoid tumours are rare, locally aggressive soft tissue growths that do not metastasise but can cause severe morbidity. They affect all age groups but are most common in young adults, particularly women. Until now, treatment has relied on surgery, radiotherapy and various systemic therapies used off-label, with limited effectiveness.
About Sarcoma UK: Sarcoma UK is the only cancer charity in the UK focusing specifically on sarcoma. We provide information and support to anyone affected by sarcoma, fund research into better treatments and campaign for better services and outcomes for sarcoma patients.
