Actelion Canada Pharmaceuticals Inc.'s Zavesca(R) (Miglustat) Approved for Reimbursement Under the Ontario Public Drug Program

LAVAL, CANADA--(Marketwire - November 09, 2010) - Actelion Canada Pharmaceuticals Inc. (SIX: ATLN) announced today that Zavesca® (miglustat) has been approved for reimbursement under the Ontario Public Drug Program. The reimbursement of Zavesca® for patients suffering from Gaucher Type 1 or Niemann-Pick type C diseases will be considered through the Exceptional Access Program according to specific clinical criteria and has been effective since September 9, 2010.

Zavesca® is indicated in Canada for the treatment of adult patients with mild to moderate Type 1 Gaucher disease for whom enzyme replacement therapy is not a therapeutic option (e.g. due to constraints such as allergy, hypersensitivity or poor venous access). Zavesca® is also indicated for the treatment of progressive neurological manifestations in adult patients and pediatric patients with Niemann-Pick type C disease.

Julian Raiman, M.D., Toronto, commented: “On behalf of the NP-C families I care for, I welcome the recent decisions of Health Canada to approve miglustat, and the Ontario Government’s move to include this drug in the Drugs for Rare Diseases Program. Allowing easier access to this medication and continuing to address the provision of novel therapies for rare diseases will not only result in better health, but it will also bring a new sense of hope to Canadian patients.”

Christine White, President, National Gaucher Foundation of Canada commented: “The recent announcement that Zavesca (miglustat) will be funded under the ODB Exceptional Access program for Ontario patients with Type 1 Gaucher disease is wonderful news. This means that patients who meet the clinical criteria now have more options in disease management.”

Tammy Vaughan, Chair of the Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF) commented: “As the first-ever approved treatment for Niemann-Pick Disease Type C, Zavesca provides relief for families and patients who are desperate to increase life expectancy and improve the quality of their lives. Including Zavesca within the remit of Ontario’s governmental reimbursement policy is a tremendous step forward in helping the cause of these patients and families. As the Chair of the Canadian Chapter of the National Niemann-Pick Disease Foundation (CCNNPDF), I hope other provinces might come to the same decision.”

Gaucher Type 1 is an inborn error of metabolism which results in a disease due to an enzyme defect. This defect leads to the progressive accumulation of glucocerebroside in the spleen, liver and lymph nodes. The usual first sign of the disease is enlargement of the spleen (located in the upper left abdomen). Other common signs include anemia, a decrease in blood platelets (clotting cells), increased pigmentation of the skin, and a yellow fatty spot on the white of the eye (called a pinguecula). Severe bone involvement is prominent and can lead to pain and collapse of the bone of the hips, shoulders, and spine.

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 states 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.

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 in the European Union, the United States, Canada, Switzerland, Brazil, Australia, Turkey, Israel, South Korea, New Zealand and Russia.

Zavesca® is indicated to slow the progression of some of the neurological manifestations in patients with Niemann-Pick type C disease.

About 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 or 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.

About Actelion

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, 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 2,400 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®)