Nirogacestat for treating desmoid tumours

Nirogacestat is in clinical development for the treatment of desmoid tumours (DT). DT is a rare condition called an intermediate tumour, which sit somewhere between non-cancerous and cancerous tumours. These types of tumours have the capacity to come back at or near the original tumour, but they do not spread. The main symptom of DT is a lump, commonly found on the arms, legs or abdomen. It can also develop in the head and neck area. There is currently no standard of care for DT, however existing treatment options include active surveillance, surgery, off-label systemic therapy and locoregional therapy. Current treatments have a risk of recurrence depending on patient age, tumour location, size and morbidity associated with the tumour. Off-label systemic therapies used in DT management either have not been studied specifically in DT or have not directly demonstrated significant improvement in patient symptom burden, functioning or overall quality of life (QoL). Thus there