ALLSCHWIL/BASEL, Switzerland, March 3, 2003 (PRIMEZONE) -- Actelion Ltd. (SWX:ATLN) (Other OTC:ALIOF) today announced that the company has started the introduction of Zavesca(R) in the European Union.
The first oral treatment for type-1 Gaucher disease is now available to patients in the U.K. In addition, a named-patient program allows commercial access to the drug through international pharmacies, if so permitted by local regulations.
Zavesca(R) (miglustat) is a small molecule developed by Oxford Glycoscience (OGS) for the treatment of patients with mild to moderate type-1 Gaucher disease for whom Enzyme Replacement Therapy (ERT) is unsuitable. Actelion expects to make the drug available throughout the European Union in the coming months.
Actelion is the license holder for Zavesca(R) in all territories worldwide, with the exception of Israel. Actelion is responsible for all regulatory and marketing activities and will book sales of Zavesca(R).
NDA amendment to be filed with the U.S. Food and Drug Administration (FDA)
In the United States, the two companies are in the final steps of filing an amendment to an existing New Drug Application (NDA). Actelion also intends to file for registration of Zavesca(R) in Japan and will evaluate opportunities in other territories, as appropriate. In Israel, license holder Teva has also started regulatory procedures for the product.
Actelion and OGS to further develop Zavesca(R)
The two companies, through a joint steering committee, will evaluate further potential uses of the product in other diseases, such as type-3 Gaucher's disease, Niemann-Pick type-C and late onset Tay-Sachs disease.
Note to the editor:
About Gaucher's disease
Gaucher's disease is a rare genetic disorder, which results from reduced activity of glucocerebrosidase, an key enzyme responsible for the metabolism of glycosphingolipids (GSL -- a subclass of fats). Symptoms include enlargement of spleen and liver, bone disease, anaemia, intense fatigue, and in some cases lung involvement.
Treating Gaucher's disease with Zavesca(R)
So far, the treatment of Gaucher's disease was either symptomatic or through replacement of the missing enzyme. Zavesca(R) is an oral inhibitor of glucosylceramide synthase, a key enzyme involved in GSL biosynthesis. The rationale for the use of Zavesca(R) is to help balance the overall level of GSLs by reducing their production or synthesis, therefore allowing the residual patient's enzyme to take care of the remaining substrate -- termed "substrate reduction."
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) has been granted orphan medicinal product status in Europe, allowing for a ten-year marketing exclusivity period following approval in Europe. A copy of the approved label for Zavesca(R) (in English language) can be found at 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).
Actelion Media & Analyst Day, 4 March 2003
Actelion Ltd. will hold an Analyst & Media day on March 4, 2003 to comment further on its financial performance, its marketing efforts for Tracleer(R) and Zavesca(R) and its substantial clinical program with four compounds in numerous indications.
For further information about attending in person or following the event via webcast, please visit www.actelion.com