Miglustat (Zavesca) may be approved in US to treat Niemann-Pick type C disease

Miglustat (Zavesca) could be approved in US to treat Niemann-Pick type C disease, a fatal neurodegenerative disease.

Miglustat (Zavesca) has received positive vote from United States Food and Drug Administration’s Endocrinologic and Metabolic Drugs Advisory Committee for the treatment of Niemann-Pick type C disease, the developer of the drug Actelion said in a press release.

The benefit/risk profile of miglustat (Zavesca) supports its approval for the treatment of progressive neurological manifestations in adult patients and pediatric patients with Niemann-Pick type C (NP-C) disease.

US FDA is currently reviewing results from the clinical trial OGT 918-007 and two multicenter NP-C disease cohort studies as well as other clinical trials using miglustat (Zavesca) in related lysosomal storage disorders for the safety and tolerability evaluation.

“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,” stated Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion.

US FDA often seeks the advice of an Advisory Committee when evaluating potential treatments for diseases. However, US FDA is not bound by the committee’s guidance.

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

What is Niemann-Pick type C disease?

NP-C disease is a very rare, relentlessly progressive and eventually fatal neurodegenerative genetic disorder for which no specific treatment is currently approved in the US.

NP-C disease primarily affects 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.

Miglustat (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.

Miglustat (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.

Zavesca pills, which contains 100 mg miglustat, is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. It is approved for this indication in the European Union, the United States, Canada, Switzerland, Brazil, Australia, Turkey, Israel, South Korea, New Zealand and Russia.

In the European Union, South Korea, Brazil and Russia, Zavesca® is also indicated for the treatment of progressive neurological manifestations in adult patients and pediatric patients with Niemann-Pick type C disease.

Actelion Ltd is a biopharmaceutical company with its corporate headquarters in Allschwil/Basel, Switzerland.

Actelion’s first drug Tracleer, an orally available dual endothelin receptor antagonist, has been approved as a therapy for pulmonary arterial hypertension. Actelion markets Tracleer through its own subsidiaries in key markets worldwide, including the United States (based in South San Francisco), the European Union, Japan, Canada, Australia and Switzerland.
Actelion is a leading player in endothelium – the single layer of cells separating every blood vessel from the blood stream.