Actelion's Zavesca cleared for NPC
Swiss drugmaker Actelion's Zavesca (miglustat) has been approved in the European Union for the treatment of progressive neurological manifestations in adult and pediatric patients with Niemann-Pick type C disease.
The agent is the first treatment to be approved for the condition, a very rare, invariably progressive and eventually fatal neurodegenerative genetic disorder. Zavesca is already indicated for patients with mild-to-moderate type 1 Gaucher disease unsuitable for enzyme replacement therapy.
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