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news.A European Medicines Agency committee has recommended orphan medicinal product designation for Amicus Therapeutics' Amigal for the treatment of Fabry disease.
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Fabry disease is a lysosomal storage disorder caused by an enzyme deficiency that leads to pain, kidney failure, and an increased risk of heart attack and stroke. Amigal is currently being studied in phase II clinical trials in the US, the UK, Brazil, and Australia.
Fabry disease affects approximately 5,000 to 10,000 people in the developed world, but recent evidence suggests that the disease may be significantly under-diagnosed.
Orphan designation granted by the EMEA provides 10 years of market exclusivity after approval and other incentives to support and encourage development of drugs for diseases that affect fewer than five in every 10,000 people in the European Union.
In addition, for diseases with an approved therapy in Europe, such as Fabry disease, a new therapy must have the potential to provide significant benefit over the existing therapy in order to receive orphan designation.
In February 2004, the FDA granted orphan designation for Amigal in the US.