European approval for treatment in fight against rare Fabry disease

TKT Europe-5S has announced that ReplagalT (agalsidase alfa) has received full marketing authorisation in the EU for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease. ReplagalT is the first product to be approved for the treatment of Fabry disease.

ReplagalT is the first product licensed under new EU legislation to develop drugs for patients suffering from rare diseases — so-called orphan medicinal products. The European Commission agreed this new legislation in the spring of last year, offering market exclusivity for 10 years for such drugs.

Fabry disease is a rare, genetic disorder that leads to premature death of affected males (on average in the patient's 40s) from renal, cardiac or cerebrovascular complications. In early childhood, patients experience severe, debilitating pain and develop skin lesions (angiokeratomas).

Later in life, patients develop kidney disease, which often progresses to renal failure, heart disease and strokes. Although females generally have a less severe clinical course and prognosis, they may experience some symptoms as severe as those of affected male patients.

'Patients affected by Fabry disease have waited a long time for a therapy that works and I am confident that ReplagalT offers a clinically effective treatment,' said Professor Michael Beck from the University Hospital in Mainz, Germany, one of the leading experts on Fabry disease in Europe.

The product is administered as a short infusion every two weeks, with the patient able to receive treatment at home.

Bo Ahlstrand, ceo of TKT Europe-5S, said, 'The safety and ease of use of ReplagalT will be important factors for patients and physicians when choosing a treatment for Fabry disease.

'We have been preparing for the approval of ReplagalT in Europe since the formation of our company over a year ago. The benefits of ReplagalT, together with our dedicated and experienced team, make us confident of the success of this landmark product in Europe.'

TKT Europe-5S is a pharmaceutical company based in Stockholm, Sweden, specialising in products for rare genetic diseases in Europe.