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.

The drug's approval is based on data from one clinical trial OGT918-007  and two multicenter retrospective cohort studies in patients with NPC.  In the trial OGT918-007, adult and juvenile patients with NPC were  randomized to either miglustat 200mg or standard of care for 12 months.  Horizontal saccadic eye movement velocity was the primary endpoint. At  12 months, HSEM velocity had improved in patients treated with  miglustat versus SOC, and results were significant when patients taking  benzodiazepines were excluded (p=0.028).