Shire of the UK is on track to launch its drug to treat the rare genetic disorder Hunter syndrome in Europe next year, after securing a positive opinion from the Committee for Medicinal Products for Human Use (CHMP).
The new product, Elaprase (idursulfase), was cleared for the same indication in the USA earlier this year and is one of five new products at Shire that together should bring in sales of $1 billion or more by 2008, accounting for more than 40% of the company's sales and removing its current reliance on attention deficit hyperactivity drug Adderall XR (mixed amphetamine salts).
Hunter syndrome is also known as mucopolysaccharidosis type II and is an enzyme efficiency disorder that causes mucopolysaccharide to build up and deposit in the tissues. It is characterised by growth problems, facial deformity and abnormal organ function and can also cause mental retardation, and in severe cases patients do not tend to live beyond age 20.
Early access to the treatment has been granted to patients with Hunter syndrome in a number of European countries including Italy, Germany, Spain, France, Sweden, Denmark and Norway, on a compassionate-use basis.
Shire estimates that there are approximately 2,000 patients worldwide afflicted with Hunter Syndrome in countries where reimbursement may be possible.