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Actelion Pharmaceuticals Ltd (CH) - Actelion receives FDA complete response letter for Zavesca (miglustat) for the treatment of Niemann-Pick type C disease

ALLSCHWIL/BASEL, SWITZERLAND - 09 March 2010 - Actelion Ltd (SIX: ATLN) announced today that the company has received a complete response letter from the U.S. Food and Drug Administration (FDA) for its supplemental New Drug Application (sNDA) for Zavesca® (miglustat) for the treatment of progressive neurological manifestations in adult and pediatric patients with Niemann-Pick type C (NP-C) disease, a rare, neurodegenerative genetic disorder.

The FDA has requested additional preclinical and clinical information. Actelion will work diligently with the FDA to explore the best ways to address the points raised by the agency.

Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented: "We remain committed to bringing an approved treatment to patients suffering from NP-C disease and in this spirit we will continue the dialogue with the FDA."

About Niemann-Pick type C disease NP-C disease is a 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 the condition. There is no specific drug therapy approved in the United States to treat NP-C disease.

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Notes to the editor

About Zavesca® (miglustat) Zavesca® (100 mg miglustat capsule) is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Zavesca® may only be used in the treatment of type 1 Gaucher patients for whom enzyme replacement therapy is unsuitable or is not a therapeutic option. 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, Russia, Australia and Canada, Zavesca® is also indicated for the treatment of progressive neurological manifestations in adult and pediatric patients with Niemann-Pick type C disease.

Zavesca® safety information In clinical studies, the most common adverse events due to Zavesca® included weight loss, diarrhea, and tremor. Other common adverse reactions were flatulence, abdominal pain, headache, and influenza-like symptoms. The most common serious adverse reaction was peripheral neuropathy. Patients should undergo neurological examination at the start of treatment and every 6 months thereafter; Zavesca® should be reassessed in patients who develop symptoms of peripheral neuropathy. Zavesca® should not be used in pregnant woman. Men should maintain reliable contraceptive methods while taking Zavesca® and for 3 months after discontinuing treatment.

Actelion Ltd 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, founded in late 1997, is a leading player in innovative science related to the endothelium - the single layer of cells separating every blood vessel from the blood stream. Actelion's over 2,300 employees focus on the discovery, development and marketing of innovative drugs for significant unmet medical needs. Actelion shares are traded on the SIX Swiss Exchange (ticker symbol: ATLN) as part of the Swiss blue-chip index SMI (Swiss Market Index SMI®).

For further information please contact:

Roland Haefeli

Vice President, Head of Investor Relations & Public Affairs

Actelion Pharmaceuticals Ltd, Gewerbestrasse 16, CH-4123 Allschwil

+41 61 565 62 62

+1 650 624 69 36

http://www.actelion.com<http://www.actelion.com/>

 

Press Release PDF<http://hugin.info/131801/R/1392243/349854.pdf>

Posted: March 2010

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