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news.The FDA's Endocrinologic and Metabolic Drugs Advisory Committee has voted favourably in question that the benefit/risk profile of Actelion's Zavesca (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.
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The decision was based on results from the clinical trial, OGT 918-007, and two multicenter NP-C disease cohort studies and clinical trials in related lysosomal storage disorders for the safety and tolerability evaluation.
Jean-Paul Clozel, MD and chief executive officer of Actelion, said: “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 Zavesca 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.”
Reportedly, Zavesca is the only specific treatment available for patients with NP-C disease. It received approval in the EU and other countries in 2009. Zavesca 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.