EU grants first orphan drug authorisations
The first marketing authorisations to be awarded under
special European provisions for orphan medicinal products have been granted.
Two products, Fabrazyme and Replagal, both of which
contain a-galactosidase-A, have been licensed to treat Fabry’s disease,
a rare inherited metabolic disorder affecting fewer than 5,000 people
patients across the world.
Fabry’s disease, a fat storage disorder also known
as angiokeratoma corporis diffusum universale, is characterised by the
absence from the body of galactosidase-A, which cleaves galactose from
lipids. Deficiency of this enzyme allows lipid accumulation in many organs
and prevents their proper function. Patients characteristically have burning
sensations in their hands and feet exacerbated by exercise and hot weather.
Heart and kidney disease is common.
Fabrazyme and Replagal have been developed independently
by TKT Europe-5S AB and Genzyme BV. They were approved under the special
procedures agreed last year for orphan medicines. As such, they will enjoy
10 years’ market exclusivity.
The orphan drug rules aim to promote the development
of medicines for patients suffering from diseases affecting fewer than
5 per 10,000 of the EU population, where no satisfactory treatment already
exists.
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