Merck has announced that NICE has issued final draft guidance recommending Ogsiveo for adults in England with progressing desmoid tumours who require systemic treatment. Eligible patients will gain immediate access through interim Cancer Drugs Fund funding.
Dr 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.”
He added: “This represents a significant forward step that meets a longstanding unmet need for patients with this challenging condition.”
Desmoid tumours are rare soft tissue tumours with an annual incidence of 2–4 cases per million people. In the UK, an estimated 340–410 new cases occur each year.
Symptoms vary by tumour location and may include pain, swelling, reduced mobility and fatigue. For some people, the condition can significantly affect mental well-being.
“Desmoid tumours are described in clinical terms as non-malignant, however, for people living with them, they are anything but benign. The World Health Organisation classifies them as intermediate tumours as they don’t fit traditional benign or malignant classifications. They often take over their lives, at a time when people are building careers, relationships and families,” said Jacqui Black, founder of Desmoid Aid UK. “They require life-long monitoring and cause chronic pain, visible disfigurement and restricted mobility.”
Nirogacestat is supported by more than four years of efficacy and safety data, including results from the phase 3 DeFi trial in 142 adults. The study showed tumour reduction and improvements in pain and quality of life, with adverse events consistent with the known safety profile.
Adam Stay, Country Lead Rare Tumours, Merck UK and Ireland, said: “We are delighted to reach this important milestone for people living with desmoid tumours in England. Patients can face years of uncertainty, recurrence and treatment burden, often with few effective options available.
“The approval of nirogacestat, the first approved systemic therapy for desmoid tumours, represents a meaningful advance in care and underscores our commitment to delivering innovative medicines that make a tangible difference to patients’ lives.”
