CRANBURY, N.J., April 11 (UPI) -- Amicus said Tuesday a European Medicines Agency committee recommended orphan designation for Amigal, a potential treatment for Fabry disease.
The EMEA's Committee for Orphan Medicinal Products made the recommendation, which now goes to the European Commission for approval.
The Food and Drug Administration granted orphan designation for Amigal in 2004.
Amigal, which Amicus is developing as an oral therapy for Fabry disease, is currently in phase 2 studies in the United States, the United Kingdom, Brazil and Australia.
Approximately 5,000 to 10,000 people in the developed world are afflicted with Fabry disease, which arises dues to an enzyme deficiency and can result in pain, kidney failure and increased risk of heart attack and stroke.
