Nirogacestat for treating desmoid tumours

Nirogacestat is in clinical development for the treatment of desmoid tumours (DT). DT are rare types of tumours , 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 and current treatments have a risk of the tumour coming back depending on patient age, tumour location, size and morbidity associated with the tumour. Treatments for DT are often “off-label” and therefore have not been approved for the treatment of DT specifically. Thus, there is a high unmet need for effective treatments that specifically target DT and improve QoL.