Actelion receives recommendation to file for US market approval
Allschwil – The FDA's Endocrinologic and Metabolic Drugs Advisory Committee has recommended (10 for, 3 against) approval for Actelion’s miglustat for the treatment of children and adults with Niemann-Pick Type C disease(NP-C), a fat storage disease that is accompanied by progressive neurological manifestations. The decision is based on extensive results from clinical trials in related lysosomal storage disorders. Since last year Zavesca(R) has already been available for European patients with NP-C disease. The drug is also indicated in the US, the EU, and other countries for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease for whom enzyme replacement therapy is unsuitable, or when it is not a therapeutic option. The FDA decision for the supplemental new drug application is expected for March 2010.