Vamorolone is currently in clinical development for the treatment of boys with Duchenne muscular dystrophy (DMD). DMD is a rare genetic, muscle wasting disease caused by a change (mutation) in the X chromosome that results in loss of the production of a protein called dystrophin. The disease primarily affects boys with symptoms including difficulty walking beginning to develop from the age of 3. As the disease progresses, muscle wastage becomes more severe resulting in increased levels of disability and serious, life threatening complications such as disease of the heart muscle and breathing difficulties. There is an unmet need as current treatments involving high-dose steroids have significant side effects that affect the patient’s quality of life.
Vamorolone is a new type of oral anti-inflammatory medicinal product that works by blocking a pathway known as NFkB, which is known to be associated with DMD symptoms. Vamorolone is designed to work in a different way to steroids resulting in the same treatment efficacy but with fewer side effects compared to steroids. If licenced, vamorolone will offer an additional treatment option for male paediatric patients with DMD aged 4 years and older.