Update: Actelion starts roll-out of Gaucher drug Zavesca in EU

Actelion has launched its oral treatment for type 1 Gaucher disease,Zavesca (miglustat), in the UK, its first world market. The small-molecule drug, which becomes the first orally-active treatment for Gaucher disease to be commercialized, is indicated for patients with mild-to-moderate type 1 disease for whom enzyme replacement therapy is unsuitable and should roll out across the rest of the European Union in the coming months.

At present, the only available therapy for Gaucher disease is Genzyme's injectable Cerezyme (imiglucerase) product, which requires twice-monthly intravenous infusion. The disease is an inherited enzyme-deficiency disorder characterized by anemia, bleeding, bone pain, enlarged spleen and liver and bone fractures.

A spokesperson for Actelion's UK subsidiary told the Marketletter that pricing for the drug has been set at L4,480 ($7,072) for a 28-day pack of Zavesca at ex-factory prices. This translates to an annual treatment cost of L58,240 per patient, she noted. While it is hard to estimate the annual cost of enzyme replacement therapy, it has been suggested that, at the lowest approved dose of 15 International Units/kg for an average 60kg patient, this comes in at around L70,000 a year. Actelion has said that the average wholesale price for Zavesca across Europe will be 90,000 euros ($98,000) per patient per year.