Benzinga | Jul 21, 2021 07:30AM EDT

Inozyme Pharma Receives Orphan Drug Designation for INZ-701 from the European Medicines Agency for the Treatment of ABCC6 Deficiency

Inozyme Pharma, Inc. (NASDAQ:INZY), a rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced that the European Medicines Agency (EMA) has granted Orphan Drug Designation to INZ-701 for the treatment of ABCC6 Deficiency. INZ-701, an investigational enzyme replacement therapy (ERT), was granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) and the EMA for the treatment of ENPP1 Deficiency.

ABCC6 Deficiency is a rare, inherited disorder that can present as generalized arterial calcification of infancy (GACI) type 2 in infants and as pseudoxanthoma elasticum (PXE) in children and adults. It is one of several disorders that features a significant decrease in plasma pyrophosphate (PPi) levels, a potent regulator of mineralization. In patients with ABCC6 Deficiency, the abnormal calcification caused by low PPi can result in vision loss and life-threatening cardiovascular complications, among other morbidities. There is no approved treatment for ABCC6 Deficiency.

"Obtaining Orphan Drug Designation from the EMA for the treatment of ABCC6 Deficiency marks another important step toward providing relief for patients who currently have no treatment options and positions INZ-701 to become the first-ever available therapy for this condition," said Axel Bolte, MSc, MA, co-founder, president, and chief executive officer of Inozyme.

Orphan Drug Designation in the European Union (EU) is granted by the European Commission based on a positive opinion issued by the EMA Committee for Orphan Medicinal Products (COMP). To qualify, a therapeutic candidate must be intended to treat a serious condition that affects fewer than five in 10,000 people in the EU, and there must be sufficient data to suggest the candidate may produce clinically relevant outcomes. The designation could allow for drug development incentives, including clinical protocol assistance, access to the centralized authorization procedure, reduced regulatory fees, and a ten-year period of market exclusivity in the EU after product approval.