Miglustat (Zavesca(R)) Receives Positive Opinion From The CHMP For Human Use For An Extension Of Indication For Niemann-Pick Type C Disease

Main Category: Neurology / Neuroscience
Also Included In: Pediatrics / Children's Health;  Regulatory Affairs / Drug Approvals
Article Date: 24 Dec 2008 - 2:00 PDT

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Actelion Ltd (SIX: ATLN) announced that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for an extension of indication for the use of miglustat (Zavesca®) in patients suffering from Niemann-Pick type C (NPC), a very rare neurodegenerative genetic disease.

Based on the CHMP opinion, the European Commission is expected to make a final decision within two months.

Actelion is currently evaluating the possibility for submissions for miglustat (Zavesca®) in NPC in other countries outside the European Union.

Zavesca® (100 mg miglustat) is already indicated for the oral treatment of 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.

About Niemann-Pick type C disease

NPC is a rare, fatal, neurodegenerative, genetic condition primarily affecting children and teenagers, but which can strike at any age. The symptoms are caused by the storage of some glycosphingolipids within certain cells in the body, including the brain. It is invariably progressive and most patients die within five to ten years of diagnosis. Neurological deterioration is a 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 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 lengthy due to its rarity and heterogeneity. There is currently no treatment option approved for this condition.

About Zavesca® (100 mg miglustat) and type 1 Gaucher disease

Zavesca® (100 mg miglustat) is indicated for the oral treatment of 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. It is approved in the European Union, United States, Canada, Switzerland, Brazil, Australia, Turkey and Israel.

Zavesca® safety information

Gastrointestinal events, mainly diarrhea, have been observed in more than 80% of patients treated with Zavesca®, either at the onset of treatment or intermittently during treatment. The majority of cases are mild and are expected to resolve after the first weeks on therapy. In clinical practice, diarrhea has been observed to respond to diet modification (reduction of lactose and other carbohydrate intake), to taking Zavesca® away from meals, and/or to antidiarrheal medicinal products such as loperamide. In some patients, temporary dose reduction may be necessary. Patients with chronic diarrhea or other persistent gastrointestinal events that do not respond to these interventions should be investigated according to clinical practice. Zavesca® has not been evaluated in patients with a history of significant gastrointestinal disease, including inflammatory bowel disease.

Peripheral neuropathy has been reported in type 1 Gaucher patients treated with Zavesca®. Peripheral neuropathy seems to be more common in patients with type 1 Gaucher disease compared to the general population. All patients should undergo baseline and repeat neurological evaluation. Patients who develop symptoms such as numbness and tingling should have a careful re assessment of risk benefit.

Zavesca® may cause fetal harm if administered to a pregnant woman and is contraindicated in women who are or who may become pregnant; patients should be informed of the potential hazard to the fetus. There is a risk of impaired fertility in men. Men should maintain reliable contraceptive methods and not plan to conceive while taking Zavesca® and for three months thereafter.

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 1800 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).

Roland Haefeli
Vice President, Head of Investor Relations & Public Affairs
Actelion Pharmaceuticals Ltd, Gewerbestrasse 16, CH-4123 Allschwil
Actelion Ltd

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