The U.S. Food and Drug Administration (FDA) and European Medicines Agency’s (EMA) Committee for Orphan Medical Products (COMP) granted an orphan drug designation to Inozyme Pharma for their product INZ-701 for the treatment of ENPP1 deficiency, a serious, life-threatening calcification disorder.
The ENPP1 gene produces a critical enzyme called ectonucleotide pyrophosphatase/ phosphodiesterase 1 (ENPP1), which regulates inorganic pyrophosphate (PPi) levels in plasma. PPi is essential for preventing harmful soft tissue calcification and for regulating normal bone mineralization. ENPP1 Deficiency manifests as either generalized arterial calcification of infancy (GACI) type 1 or autosomal recessive hypophosphatemic rickets type 2 (ARHR2). GACI type 1 is a devastating and often fatal disease affecting infants and is characterized by calcification and narrowing of large and medium-sized arteries, resulting in heart failure and death in about half of patients within the first six months of life. ARHR2 manifests in the post-infancy stage and causes rickets, weakened bones, repeated bone fractures, skeletal deformities, short stature, muscle weakness, fatigue, and bone pain.
INZ-701 is an enzyme replacement therapy developed by Inozyme Pharma intended for the treatment of calcification disorders of the circulatory system, bones, and kidneys. The therapy has exhibited the potential to generate plasma pyrophosphate (PPi) and to restore it to appropriate physiological levels, thus preventing calcification in the vasculature and kidneys and normalizing bone.
“Orphan Drug Designation, both in the United States and the European Union, is an important regulatory milestone for Inozyme as we continue our quest to develop INZ-701 for patients with rare and life-threatening calcification disorders,” said Axel Bolte, co-founder and chief executive officer of Inozyme, in a recent statement. “The dual designations from the FDA and EMA provide crucial momentum for INZ-701, putting us in an excellent position to rapidly advance the clinical development program for this novel enzyme replacement therapy.”