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Pulmonary Arterial Hypertension
Gaucher Disease
Actelion is a biopharmaceutical company with its corporate headquarters in Allschwil/Basel, Switzerland. Actelion was founded in 1997 and its shares have been listed on the SWX Swiss Exchange with the ticker symbol ALTN since 2000. The company is focused on the discovery, development and commercialisation of synthetic, small-molecule drugs as innovative treatments to serve high unmet medical needs. Small-molecule drugs are more readily made suitable for oral ingestion than are biologicals. In addition, unlike the larger proteins, smallmolecule drugs can be capable of crossing the blood/brain barrier. This ability is important in Actelion's efforts to develop treatments for certain neurological ailments.
Actelion has subsidiaries in 21 countries including the United States, Canada, Brazil, Australia, Japan, Switzerland and a number of EU countries.
Actelion's lead product, Tracleer® (bosentan), is the first commercially available oral dual endothelin receptor antagonist and is currently approved for the treatment of pulmonary arterial hypertension (PAH), a rare chronic lifethreatening disorder that severely compromises the function of the lungs and heart. Tracleer® has been approved for the treatment of PAH in more than 30 countries. Actelion markets Tracleer® through it own subsidiaries in key markets including, the United States and the European Union, as well as Australia, Canada, Japan and Switzerland.
Actelion's second marketed product, Zavesca® (miglustat), is currently the only approved oral treatment for type 1 Gaucher's disease, a rare and debilitating metabolic disorder, and is indicated in patients for whom enzyme replacement therapy is unsuitable. Zavesca® was in-licensed from Oxford Glycoscience in 2002 and Actelion has now been granted marketing rights throughout the world, except in Israel. The product was launched in the EU in March 2003 and in the US in Jan 2004.
Clinical Development Following the success of Tracleer® (bosentan) in pulmonary arterial hypertension (PAH), Actelion is also developing bosentan for the treatment of other conditions including digital ulcers, pulmonary fibrosis, chronic thromboembolic
pulmonary hypertension, sickle cell disease related to PAH, and metastatic melanoma.
Zavesca® (miglustat) is currently being developed to treat several other lysosomal- storage disorders such as type 3 Gaucher's disease and Niemann-Pick type C.
Actelion's late-stage product candidates include clazosentan, an intravenous endothelin receptor in Phase IIb/III clinical trials for the treatment of complications of subarachoid haemorrhage.
Early-stage projects are the undisclosed Actelion-1, in development for cardiovascular indications, and an orexin receptor antagonist developed for sleep disorders.
Drug Discovery
Actelion's research team has discovered renin inhibitors with high level of oral bioavailability. The expected therapeutic application for a renin inhibitor is hypertension. With the large potential, size and complexity of clinical trials, an alliance with Merck & Co to discover, develop and commercialise oral rennin inhibitors has been formed. Actelion has also discovered several potent and orally active orexin receptor antagonists. With one of these compounds Actelion initiated a clinical program in 2005, evaluating its efficacy in sleep disorders.
An S1P1 agonist is tested for its potential in immunological indications.
Beta-secretase (BACE-1) inhibitors are evaluated for their potential use in prophylaxis and treatment of Alzheimer's disease.
Actelion is discovering several antibiotics targeting resistant bacteria.
In addition, yet unnamed compounds (Actelion-3 to -11) are in full preclinical development for cardiovascular, onco/immuno, cns and antiinfective indications.
Since its foundation in 1997, Actelion has filed more than 100 priority patent applications.
Useful links:
- Actelion
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Company Info
