The EMEA orphan drug designation provides incentives to companies that develop drugs for diseases affecting no more than 50 in 100,000 persons in the European Union, or drugs intended for a life-threatening, seriously debilitating, or serious and chronic condition, and for which there is no satisfactory authorized therapy or where the drug will provide a significant benefit.
The designation can potentially provide a drug 10 year EU market exclusivity for a particular indication. In addition, orphan drug status provides regulatory assistance, reduced regulatory fees associated with applying for marketing approval, and protocol assistance.
The company announced in May that the Food and Drug Administration had granted Troxatyl orphan drug designation in the US for the treatment of the disease.