Merck (known as MDS outside the US and Canada) has announced that Ogsiveo (nirogacestat) has been recommended by the National Institute for Health and Care Excellence (NICE) to treat adults with progressing desmoid tumours who require systemic treatment.
Desmoid tumours, also known as desmoid-type fibromatosis, are rare soft tissue tumours that have a reported annual incidence of approximately 2-4 cases per million people. In the UK, there are an estimated 340-410 new cases each year. They predominantly occur in adults aged 30-40 years and are more common in women.
Ogsiveo is the first and only systemic therapy approved in England to treat this invasive and often debilitating condition. In the phase 3 DeFi study, Ogsiveo demonstrated tumour reduction, as well as improvements in pain and quality of life.
Adam Stay, Country Lead Rare Tumours, Merck UK and Ireland, said: “Patients can face years of uncertainty, recurrence and treatment burden, often with few effective options available. The approval of Ogsiveo, the first approved systemic therapy for desmoid tumours, represents a meaningful advance in care.”
Desmoid tumours often present as a firm or palpable lump. Symptoms vary according to the tumour location and may include pain, swelling, reduced mobility, difficulty carrying out day-to-day activities and fatigue. For some people, the pain and symptoms associated with desmoid tumours may significantly affect their mental well-being, causing anxiety, depression, fear and uncertainty about the future, as well as concerns regarding body image.
Alex Lee, Consultant Medical Oncologist, The Christie NHS Foundation Trust, said: “Desmoid tumours are rare and unpredictable chronic conditions that typically arise in early adulthood, and which can cause significant pain and debilitation that continue for years and, in some cases, can even be life-threatening.
“Treatment is often challenging, with surgery typically ineffective in preventing tumour recurrence, and systemic therapy historically confined to off-license use of drugs. The availability of nirogacestat as a treatment for progressing desmoid tumours provides clinicians with access to a targeted drug treatment of established effectiveness. This represents a significant forward step that meets a longstanding unmet need for patients with this challenging condition.”