SOURCE: Actelion Pharmaceuticals Ltd

September 08, 2009 11:45 ET

Actelion updates US Healthcare Professionals for patients with type 1 Gaucher disease - Information on the potential use of Zavesca® (miglustat) oral capsule to address the supply shortage of intravenous enzyme replacement therapy (imiglucerase)

ALLSCHWIL, SWITZERLAND--(Marketwire - September 8, 2009) -


ALLSCHWIL/BASEL, SWITZERLAND - 8 September 2009 - Actelion Ltd (SIX: ATLN) has distributed updated information to Healthcare Professionals who treat type 1 Gaucher patients in the United States regarding the potential use of Zavesca® (miglustat) to minimize the impact of the supply shortage of imiglucerase (produced and marketed by Genzyme Corporation under the brand name Cerezyme®).

Despite strict supply conservation measures currently being implemented, some patients are facing the prospect of an interruption of their imiglucerase treatment. This shortage is now expected to last longer and to affect more patients than initially predicted in June 2009 by the product manufacturer.

Accordingly, Actelion has taken all the necessary steps to ensure access to Zavesca® where this important oral therapy has been approved for use by patients with Type 1 Gaucher disease, as determined by their physicians. In the United States, Zavesca® has been available since 2003.

Following communication with the US Food and Drug Administration (FDA), Actelion has issued the following letter to remind Health Care Professionals in the United States about the availability of Zavesca® as an immediate treatment option for appropriate type 1 Gaucher patients in relation to the current imiglucerase shortage. Health Care Professionals may also receive additional information by email (usmedinfo@actelion.com) or by calling toll-free (866) 228-3546.

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Notes to the editor:

About 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 (European Union) or is not a therapeutic option (United States). It is approved in the European Union, the United States, Canada, Switzerland, Brazil, Australia, Turkey, Israel, South Korea and New Zealand.

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

About type 1 Gaucher disease

Type 1 Gaucher disease, an inherited autosomal recessive disorder, is the most common disease among all inherited glycosphingolipid storage disorders, affecting approximately 7,000 patients world-wide. It is a multi-system disease characterized by hepatomegaly, splenomegaly, anemia and thrombocytopenia. In addition, bone manifestations are often underdiagnosed and include osteopenia, lytic lesions, chronic pain, acute episodes of 'bone crisis', bone infarcts, and osteonecrosis, resulting in long-term disability. Approved treatments for patients with type 1 Gaucher disease include intravenous enzyme replacement therapy with Cerezyme® (imiglucerase) and oral substrate reduction therapy with Zavesca® (miglustat).

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.

Cases of peripheral neuropathy have been reported in patients with type 1 Gaucher disease 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 2000 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®).

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Contact Information

  • 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