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Zavesca receives positive vote from FDA for the treatment of Niemann-Pick type C disease

15 Jan 2010 - Actelion Ltd announced that United States (US) Food and Drug Administration's (FDA) Endocrinologic and Metabolic Drugs Advisory Committee voted (10 yes to 3 no) in its final vote in question that the benefit/risk profile of Zavescaź (miglustat) supports its approval for the treatment of progressive neurological manifestations in adult patients and pediatric patients with Niemann-Pick type C (NP-C) disease.
 
The decision was based on results from the clinical trial OGT 918-007 and two multicenter NP-C disease cohort studies as well as other clinical trials in related lysosomal storage disorders for the safety and tolerability evaluation.
 
Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented: "We are pleased that the Advisory Committee today recognized the importance of this therapeutic advance and voted to recommend approval of the supplemental New Drug Application (sNDA) of Zavescaź for the treatment of NP-C disease. We will continue to work closely with the FDA to facilitate the completion of the priority review of the sNDA."
 
Zavescaź is the only specific treatment available for patients with NP-C disease. It received approval in the European Union (EU) and other countries in 2009. Zavescaź 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 is not a therapeutic option. The use of Zavescaź is supported by over 10 years of clinical trials and post-marketing experience across indications.