EMA orphan drug committee makes 16 recommendations at May meeting

The European Medicines Agency’s Committee for Orphan Medicinal Products (COMP) adopted a total of 16 recommendations for orphan designation during its May meeting. Orphan designation can be granted early in the development process to substances that are intended for diagnosis, prevention or treatment of life-threatening or very serious conditions that affect not more than five in 10,000 people in the European Union or that would not be developed without incentives.

One of these diseases for which no adequate treatment currently exists is infection-associated hemolytic uremic syndrome (HUS). During its meeting, the COMP recommended granting an orphan designation for eculizumab (from US drugmaker Alexion Pharmaceuticals) used for the treatment of this severe, life-threatening complication of enteric infections with Shiga toxin-producing Escherichia coli bacteria, such as enterohemorrhagic E. coli, also called EHEC.

Infection-associated HUS occurs predominantly in childhood. Although it is a very rare disease, estimated to occur in less than 0.07 per 10.000 patients a year, infection-associated HUS is recognized as the most common cause of acute kidney failure in infants and young children. It can also affect adults and the elderly, who often die as a result of the disease.