Zavesca(r) Capsules Available for Prescription in the U.S.
First Oral Treatment Option for Type 1 Gaucher Disease
ALLSCHWIL and BASEL, Switzerland, Jan. 5, 2004 (PRIMEZONE) -- Actelion Ltd (SWX:ATLN) today announced the availability of Zavesca(r) (miglustat) capsules in the United States. Zavesca(r) is the first oral treatment option for type I Gaucher disease and is also already approved and available in the European Union.
Type 1 Gaucher disease is a rare genetic lysosomal storage disorder affecting an estimated 10,000 individuals worldwide. It is a progressive condition that is caused by a deficiency of the enzyme glucocerebrosidase.
This deficiency results in the storage of excess amounts of the glycosphingolipid (GSL) substrate, primarily in macrophages, which accumulate in the liver, spleen and bone marrow. This accumulation leads to liver and spleen enlargement/dysfunction, anemia, thrombocytopenia, bone disease and pain.
Zavesca(r) is the first in a new class of oral drugs known as substrate reduction therapy (SRT). The goal of treatment with SRT is to reduce the production of the glycosphingolipid substrate to a level that can be effectively cleared by the naturally occurring enzyme, glucocerebrosidase.
Prior to the availability of Zavesca(r), the only other FDA approved treatment was enzyme replacement therapy (ERT), which is delivered via an intravenous infusion twice monthly.
The FDA approved Zavesca(r) mid 2003 for the treatment of adult patients with mild to moderate type I 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).
In two clinical studies, Zavesca(r) significantly reduced liver and spleen volumes and increased platelet and hemoglobin levels in patients who were either naive or had discontinued ERT for at least three months. In a third study, Zavesca(r) maintained the hematological and organ volume benefits in patients who had previously been on long-term ERT (mean of 5.6 years).
Safety Information
Peripheral neuropathy has been reported in type 1 Gaucher patients treated with Zavesca(r). Patients should undergo a neurological exam at the start of treatment and every 6 months thereafter. Zavesca(r) should be reassessed in patients who develop symptoms of peripheral neuropathy. Zavesca(r) 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 apprised 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(r) and for 3 months thereafter.
Zavesca(r) Distribution in the United States
Zavesca(r) 100mg capsules are supplied in a carton of 90 capsules, packed in 5 blister cards of 18 capsules each. Zavesca(r) is available only through the single specialty pharmacy, Curascript by calling 1 866-228-3546.
Physicians interested in prescribing Zavesca(r) must sign a physician statement affirming that they have reviewed the key safety information for Zavesca(r) and are qualified to treat Gaucher disease.
Physicians in the United States seeking to prescribe or patients wanting to learn more about Zavesca(r) should call 1-866-228-3546 or visit
http://www.zavesca.com.
Background on Actelion and Zavesca(r)
Actelion is the license holder for Zavesca(r) worldwide, with the exception of Israel where the drug is also approved. Actelion is responsible for all regulatory and marketing activities and will book all sales of Zavesca(r). The drug has been in licensed from Oxford Glyco Sciences, now Celltech Group.
Through a global steering committee, Actelion and Celltech are currently conducting clinical studies with Zavesca(r) for the treatment of other lipid storage disorders, such as type 3 Gaucher disease, Niemann-Pick type C, and Late Onset Tay-Sachs.
Note to the editor:
About type 1 Gaucher disease
Type 1 Gaucher disease is a rare genetic disorder, which results from the reduced activity of glucocerebrosidase, a key enzyme responsible for the catabolism of glycosphingolipids (GSL). The deficiency of this enzyme results in the storage of excess amounts of the GSL substrate, primarily in macrophages, which accumulate in the liver, spleen and bone marrow. This accumulation leads to liver and spleen enlargement/dysfunction, anemia, thrombocytopenia, bone disease and pain. In rare cases, the lung may be involved, leading to conditions such as pulmonary hypertension.
Treating type 1 Gaucher disease with Zavesca(r)
Until the availability of Zavesca(r), the only approved treatment for type 1 Gaucher disease was replacement of the deficient enzyme by intravenous infusion (ERT). The rationale for the use of Zavesca(r) is to help balance the overall level of glycosphingolipid (GSL) substrate by reducing their synthesis, to a level that can be effectively cleared by the residual activity of the deficient enzyme. This is known as "substrate reduction therapy (SRT)".
Zavesca(r) regulatory background
In November 2002, the European Commission approved Zavesca(r), with unified labeling valid in all 15 European Union-Member States. Zavesca(r) is commercially available in the United Kingdom, Germany, Ireland, the Netherlands and Greece. In other territories at named patient program was set up to allow commercial access to the drug through international pharmacies, if so permitted by local regulations. In June 2003, Zavesca(r) was approved in Israel. In the United States, the FDA approved Zavesca(r) on 31 July 2003, following a NDA amendment filed by Actelion on 7 February 2003. A copy of the approved label for Zavesca(r) (in English language) can be found at
http://www.actelion.com or
http://www.zavesca.com
Actelion Ltd
Actelion Ltd is a biopharmaceutical company with its corporate headquarters in Allschwil/Basel, Switzerland. Actelion's first drug Tracleer(r), an orally available dual endothelin receptor antagonist, has been approved as a therapy for pulmonary arterial hypertension. Actelion markets Tracleer(r) through its own subsidiaries in key markets worldwide, including the United States (based in South San Francisco), the European Union as well as Canada 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 focuses on the discovery, development and marketing of innovative drugs for significant unmet medical needs. Actelion shares are traded on the SWX Swiss Exchange (ticker symbol: ATLN).
For further information please contact:
Actelion Ltd, Gewerbestrasse 16, CH-4123 Allschwil
Investor Contact Roland Haefeli +41-61-487-3458
+1-650-624-6936
Media Contact Peter Engel +41-61-487-3628
http://www.actelion.com
http://www.zavesca.com
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