FDA Advisory Committee Recommends Expanding Miglustat Use to Niemann-Pick Disease Type C

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 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."

Zavesca® 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. 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. The use of Zavesca® is supported by over 10 years of clinical trials and post-marketing experience across indications.

The FDA often seeks the advice of an Advisory Committee when evaluating potential treatments for diseases, for instance when there is no approved therapy available. The FDA is not bound by the committee's guidance, but takes its recommendation into consideration.

The decision of the FDA for the sNDA is expected in March 2010.

About Niemann-Pick type C disease
NP-C disease is a very rare, fatal, neurodegenerative, genetic condition, primarily affecting children and teenagers although the clinical manifestations can become apparent at any age. The symptoms are caused by the storage of some lipids, such as glycosphingolipids and cholesterol, within certain tissues in the body, including the brain. It is invariably progressive and most patients die within five to ten years of diagnosis; for the majority, the disease is fatal during childhood. Neurological deterioration is the key feature of the disease, and can manifest itself as clumsy body movements, balance problems, slow and slurred speech, difficulty in swallowing, problems with eye movements and seizures. Intellectual decline is also common. In the final stages of the disease the child or young adult is frequently bedridden, has little muscle control and is intellectually impaired. Diagnosis of the disease can be difficult and may take years due to the rarity and heterogeneity of this condition.

Source: Actelion