ERYTECH Pharma gains EU orphan designation for Enhoxy

French late-stage biopharma company ERYTECH Pharma says that the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency has adopted a positive opinion to grant Orphan Drug Designation to its investigational product Enhoxy for sickle cell disease.

Enhoxy, human erythrocytes encapsulating inositol hexaphosphate, enhances the oxygenation properties of red blood cells by reducing their oxygen-hemoglobin affinity and allowing them to release more oxygen. Hypoxia, lack of oxygen in the blood, is associated with the initiation of cell sickling and severe cell sickling crisis. Total blood exchanges, using several blood units, are often needed to reverse hypoxic conditions and prevent crises in patients with this disease. Enhoxy targets to reduce the number of blood transfusions needed.

An ODD provides ten years of market exclusivity after marketing authorization in all member states of the European Union, a streamlined regulatory review process and important fee reductions.