Results for the substance nirogacestat

Nirogacestat is an oral medicine used in adults with progressing desmoid tumours when systemic treatment is needed. Desmoid tumours usually do not spread like many cancers, but they can grow locally and cause pain, reduced movement, or pressure on nearby organs. Treatment aims to slow the disease and improve symptoms.

The medicine inhibits gamma-secretase, an enzyme involved in signalling pathways that may support tumour growth. It is generally taken twice daily, with or without food, and tablets should be swallowed whole. Do not crush, break or chew them. Tell your doctor about all prescription, over-the-counter and herbal medicines, because important interactions can occur.

Common side effects include diarrhoea, nausea, tiredness, rash, mouth inflammation, headache, abdominal pain, hair loss and blood test changes such as low phosphate or increased liver enzymes. In women who could become pregnant, ovarian function may be affected, sometimes causing menstrual changes or symptoms similar to menopause.

Clinical monitoring and blood tests are needed during treatment. Contact your healthcare team quickly if you develop severe diarrhoea, dehydration, fever, widespread rash, strong abdominal pain, or major menstrual changes. Nirogacestat can harm an unborn baby; use effective contraception and discuss fertility preservation before starting treatment.