The Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) adopted a positive opinion to grant Orphan Drug Designation (ODD) to ERYTECH Pharma's 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.

“I am delighted with this positive opinion. This ODD is an important step for the development of the Enhoxy product that, we believe, can bring significant benefit to the patients suffering from this debilitating disease. With a strong preclinical package, we are now scaling up manufacturing under cGMP to be ready to initiate clinical trials next year,” said Dr Yann Godfrin, executive vice president and chief scientific officer.

“Enhoxy is a good example of the potential of our RBC based platform to bring meaningful benefit to patients with unmet needs in oncology and rare diseases.” adds Pierre-Olivier Goineau, chief executive officer, “While we are focused on advancing the oncology franchise, indications like sickle cell disease present significant opportunity for value creation to Erytech, value we intend to capture through partnerships and alliances with therapy specialists.”

SCD is a genetic disease causing amino acid substitution on the beta-chain of haemoglobin. This pathology is characterized by abnormal blood rheology and periods of painful vascular occlusive crises.

ERYTECH Pharma SA is a late-stage French biopharmaceutical company developing medicinal products for orphan oncology and rare diseases.