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Actelion Ltd.. (4/2/12). "Press Release: Japan’s Ministry of Health, Labour and Welfare Grants Approval for Actelion’s Miglustat for the Treatment of Niemann-Pick Type C Disease". Allschwil.

Organisations Organisation Actelion Pharmaceuticals Japan Ltd.
  Group Actelion (Group)
  Organisation 2 Ministry of Health, Labor and Welfare (MHLW) (J, 2004)
  Group Japan (govt)
Product Product Zavesca®
Persons Person Tanaka, Satoshi (Actelion 201204 President of Actelion Japan + South Korea)
  Person 2 Haefeli, Roland (Actelion 201105 VP Head Investor Relations + Public Affairs)
     


Actelion Ltd (SIX: ATLN) announced today that Japan's Ministry of Health, Labour and Welfare has granted approval for miglustat for the treatment of Niemann-Pick type C disease.

The approval was based on the data generated for the first approval of miglustat in this indication in the European Union, as well as data specifically generated for the submission to the Japanese Health Authorities.

Satoshi Tanaka, President of Actelion Japan and South Korea commented: "I am very proud to have achieved this successful approval. Our organization has been able to generate the additional clinical data required for the first approval of miglustat in Japan, successfully registered the drug in this orphan disease and we are looking forward to bringing the first therapy to patients with this devastating neurodegenerative disease."

Actelion Pharmaceuticals Japan will now ensure that miglustat, marketed under the tradename Brazaves®, is made available to patients as soon as possible.

###

Notes to the editor

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.

About Miglustat

Miglustat, an orally available molecule with a large volume of distribution, is currently marketed globally under the trade name Zavesca®.

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 43 countries.

Zavesca is also indicated for the treatment of progressive neurological manifestations in adult patients and pediatric patients with Niemann-Pick type C disease. It is approved for this indication in 42 countries.

Miglustat will be marketed in Japan under the tradename Brazaves®.

Miglustat safety information

In clinical studies, the most common adverse events due to miglustat 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; miglustat should be reassessed in patients who develop symptoms of peripheral neuropathy. Miglustat should not be used in pregnant woman. Men should maintain reliable contraceptive methods while taking miglustat and for 3 months after discontinuing treatment.


About Actelion Pharmaceuticals Japan

Actelion Pharmaceuticals Japan was established in 2001 to meet the medical needs of patients with pulmonary arterial hypertension in Japan. Since its foundation the company has established a significant presence in the pharmaceutical market and also provides clinical development for the specific needs of the Japanese Health Authorities. The organization also has a central role for East Asian cross-border clinical development activities, such as in South Korea. In 2011 Actelion Pharmaceuticals Japan contributed 10% of Actelion's total product sales.


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 2500 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
Senior 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

   
Record changed: 2012-05-06

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