INZ-701 Gets Orphan Drug Designation From European Medicines Agency

Release Date: 15-Sep-2021

European Medicines Agency (EMA) has granted Orphan Drug Designation to INZ-701 for the treatment of ABCC6 Deficiency, an investigational enzyme replacement therapy (ERT), was granted Orphan Drug Designation by the US FDA and the EMA for the treatment of ENPP1 Deficiency.


INZ-701 is an ENPP1 enzyme replacement therapy (ERT) to treat the mineralization disorders of the kidneys, circulatory system, and bones. INZ-701 therapy has shown potential to make plasma pyrophosphate (PPi) and to restore it to appropriate physiological levels, thereby inhibiting calcification in the vasculature and kidneys, while at the same time improving bone abnormalities. Inozyme is preparing to initiate a Phase 1/2 clinical trial in patients with ENPP1 Deficiency and a separate Phase 1/2 clinical trial in patients with ABCC6 Deficiency in mid-2021.


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.

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