Ganaxolone for treating tuberous sclerosis complex-related epilepsy after 2 or more treatments

Ganaxolone is in clinical development for the treatment of tuberous sclerosis complex (TSC) -related epilepsy. TSC is a rare genetic condition that causes non-cancerous tumours to grow in various organs, including the brain, skin, kidneys, heart, eyes and lungs. These tumours result from changes in the TSC1 or TSC2 genes and can lead to epilepsy, a condition where the brain is prone to seizures, defined as, sudden bursts of electrical activity in the brain that disrupt normal functioning like behaviour, movement, feelings or consciousness. Approximately 50% of patients with TSC-related epilepsy become resistant to current anti-epileptic drugs, making new treatment options important.