Govorestat for treating galactosemia in people aged 2-65 years

Govorestat is in clinical development for treating classic galactosemia. Classic galactosemia is a rare disorder affecting the body’s ability to break down (metabolise) the sugar present in milk (galactose) into another type of sugar called glucose. Glucose provides a source of energy for body cells. Classic galactosemia is caused by a faulty (mutated) gene, meaning that the body can’t make an enzyme (protein) which normally breaks down galactose into glucose. When not broken down properly, galactose is converted to a toxic substance called galactitol, which builds up in cells. This causes damage to different tissues in the body, leading to complications such as juvenile cataracts and learning difficulties. Currently, classic galactosemia is treated by removing dairy products, the main source of galactose, from the diet. This can help to control symptoms in babies; however, it does not prevent long-term complications of the disease such as mental disabilities that develop later in life. There are currently no drug treatments available for patients with classic galactosemia.