According to the company, Zavesca is the first treatment to be approved for patients with Niemann-Pick type C disease, a very rare, invariably progressive and eventually fatal neurodegenerative genetic disorder affecting both children and adults.
Zavesca (100mg miglustat) is already indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Zavesca may only be used in the treatment of type 1 Gaucher patients for whom enzyme replacement therapy is unsuitable, the company said.
Jean-Paul Clozel, CEO of Actelion, said: I am very proud that Actelion – together with the scientific community – has been able to demonstrate the role of Zavesca in reducing the progression of clinically relevant neurological symptoms in patients with NPC. Actelion will continue to support the rare disease community in its efforts to advance science and medicine for the patient.