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| |  Miglustat Approved in the EU for Niemann-Pick Type C Disease NEW YORK -- January 30, 2009 -- The European Union has approved miglustat (Zavesca) for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease (NPC). "For the first time we have an approved therapy for NPC," commented Ed Wraith, MD, Royal Manchester Children's Hospital, Manchester, United Kingdom. "The data on the effects of treatment with [miglustat] obtained in a clinical trial and in a retrospective cohort study consistently showed a favorable clinical response. As a treating physician, I am acutely aware of the importance of reducing progression of neurological symptoms." The approval is based on data from clinical trial OGT918-007 -- a multicentre retrospective cohort studies of adult and juvenile patients with NPC (n=29) aged 12 years and older. Patients were randomised to either miglustat 200 mg TID (n=20) or standard of care (n=9) for 12 months. In addition, 12 children aged 4 to 12 years received miglustat at a dose adjusted for body surface area. All patients were then given miglustat for another 12 months. Horizontal saccadic eye movement (HSEM) velocity was the primary endpoint. Other endpoints included swallowing, ambulation, neurological examination, neuropsychological assessment, tremor, and quality of life. At 12 months, HSEM velocity had improved in patients treated with miglustat versus those receiving standard care; results were significant when patients taking benzodiazepines were excluded (P=.028). Children showed an improvement in HSEM velocity of similar size at 12 months. Improvement in swallowing capacity, stable auditory acuity, and a slower deterioration in ambulatory index were also seen in treated patients aged older than 12 years. In an uncontrolled extension phase of the OGT918-007 trial, data indicated that treatment with miglustat can provide disease stabilisation for important markers of neurological dysfunction in NPC disease, both in the juvenile/adult and paediatric cohorts, further strengthening the interpretation of a treatment effect of miglustat observed at 12 months in the controlled phase of the trial. Gastrointestinal events, mainly diarrhoea, have been observed in more than 80% of patients treated with miglustat, either at the onset of treatment or intermittently during treatment. The majority of cases were mild and resolve after the first weeks of therapy. Cases of peripheral neuropathy have been reported in patients treated with miglustat. Peripheral neuropathy seems to be more common in patients with type 1 Gaucher disease compared with the general population. All patients should undergo baseline and repeat neurological evaluation. In patients included in the clinical trial, 40% to 50% of patients had platelet counts below the lower limit of normal at baseline. Monitoring of platelet counts is recommended in these patients. SOURCE: Actelion Ltd
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