ALLSCHWIL/BASEL, July 31, 2003 (PRIMEZONE) -- Actelion Ltd (SWX: ATLN) today announced that the US Food and Drug Administration (FDA) has approved Zavesca(r) (miglustat) capsules the first oral treatment option for type 1 Gaucher disease.
Type 1 Gaucher disease is a rare genetic lipid storage disorder affecting an estimated 10,000 individuals worldwide. It is a progressive condition that is caused by a deficiency of glucocerbrosidase, an important enzyme in the metabolism of key lipids in the body. The deficiency of this enzyme results in accumulation of excess amounts of glycosphingolipids (GSLs) in specific cells primarily in the liver, spleen and bone marrow. Such accumulation leads to liver and spleen enlargement/dysfunction, anemia, bone disease and pain.
Current therapy includes enzyme replacement therapy (ERT), which is delivered via an intravenous infusion twice monthly.
Zavesca(r) is the first in a new class of drugs known as substrate reduction therapy (SRT), which reduces the amount of glycosphingolipid (GSL) production to a level which can effectively cleared by the naturally occurring glucocerbrosidase in the cells. Zavesca(r) will be available to patients in the United States later this year.
Zavesca(r) Indication in the US
The FDA has approved Zavesca(r) 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). Woman who are or may become pregnant should not take Zavesca(r).
Zavesca(r) is approved in the European Union and is commercially available in the United Kingdom and Germany.
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 was originally developed by Oxford GlycoSciences (OGS), now part of the Celltech Group.
Actelion and Celltech to further develop Zavesca(r)
Through a joint 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 reduced activity of glucocerebrosidase, a key enzyme responsible for the catabolism of glycosphingolipids (GSL) -- a subclass of fats stored in cells primarily found in the liver, spleen and bone. There is striking clinical variability amongst affected individuals with respect to symptoms and disease progression. Common symptoms include an enlargement/dysfunction of the spleen and liver, bone disease, anemia, and in some cases lung involvement, including 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. The rationale for the use of Zavesca(r) is to help balance the overall level of glycosphingolipids (GSLs) in the cells by reducing their synthesis, therefore allowing the patient's residual enzyme activity to effectively metabolize the remaining GSLs. This is called 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 and commercial availability in the United Kingdom and Germany. 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
Actelion Ltd Actelion Ltd is a biopharmaceutical company with its corporate headquarter 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).