EU recommends first ever therapy for Hunter syndrome
pharmafile | October 24, 2006 | News story | Sales and Marketing |
Shire has received full EU recommendation for the use of Elaprase for the long-term treatment of patients with Hunter syndrome.
Marketing authorisation is expected early in 2007 and follows US approval earlier this year.
Elaprase (idursulfase), an orphan drug, has already been granted early access in a number of European countries, including Italy, Germany, Spain, France, Sweden, Denmark and Norway and as of mid-October, 58 European patients have been prescribed the drug.
Hunter syndrome (HS) is a hereditary disease, in which the breakdown of a mucopolysaccharide (a chemical widely distributed in the body) is defective, leading to a build-up of the chemical. Patients suffer from coarse facial features, abnormal function of multiple organs and in severe cases, early death.
It is inherited as an X-linked recessive disease by women and is mainly passed on to males. Affected children may develop an early onset type (severe form) shortly after the age of two that causes a large skull, abnormal facial characteristics, profound mental retardation, spasticity, aggressive behaviour, joint stiffness and death before the age of 20. A mild form has a late onset and produces less severe symptoms.
Elaprase, delivered via weekly infusions, replaces the missing enzyme that HS patients fail to produce in sufficient quantities. The data supporting the EU application has come from the single largest, longest and most comprehensive pivotal (phase II/III) study of any lysosomal storage disease (LSD) to date.
Dr Ed Wraith, consultant paediatrician at Manchester Childrens Hospital and a specialist in mucopolysaccharidosis disorders, said: When licensed, this therapy will provide physicians with the first and only treatment for Hunter syndrome since it was first recognised over 100 years ago.
Shire estimates that there are approximately 2,000 patients worldwide afflicted with Hunter syndrome.
