EU Commission approves first Hunter syndrome therapy

The EU commission has granted marketing authorisation for the use of UK-headquartered Shire Pharmaceutical's Elaprase (idursulfase) for the long-term treatment of patients with Hunter syndrome, a progressive and rare genetic disorder.

Hunter syndrome, which affects mostly men, results in individuals who cannot produce in sufficient quantities the enzyme iduronate-2-sulfatase. The lack of the enzyme, which helps the body break down and recycle complex carbohydrates called glycosaminoclycans (GAG), creates a build up of GAG. The GAG buildup causes hearing loss, declining heart function, an enlarged liver and spleen and joint stiffness.

Hunter syndrome is classified as a lysosomal storage disorder, the symptoms of which become noticeable after two years of age. Life expectancy for the more severely affected is only 10-20 years of age.

Elaprase, which is delivered by weekly infusions, replaces the deficient or missing enzyme. Treatment leads to improved endurance as measured by the six-minute walk test, while clinical trials have shown significant improvement in spleen and liver size and urine GAG levels.

Professor Michael Beck from the University Clinic of Mainz, Germany, specialist in Mucopolysaccharidosis (MPS) disorders, said: "The safety and efficacy data from clinical studies are promising and demonstrate the potential of this therapy to completely change the approach to Hunter syndrome treatment to the overall management and control of many of the symptoms of the disease."

Matthew Emmens, CEO of Shire, said of the approval: "We are delighted with the approval of Elaprase in Europe, the first and only enzyme replacement treatment for Hunter syndrome. This decision reflects Shire's commitment not only to the development of Elaprase, but also our wider commitment to developing innovative therapies for other, little researched genetic disorders."

Marketing authorisation for Elaprase follows positive opinion issued by The Committee for Medicinal Products for Human Use (CHMP) in October 2006. The data supporting the license came from the largest and longest registration study of any lysosomal disorder. Elaprase will be launched across Europe over the next 18 months.