It was approved by the FDA in 2020 for the treatment of primary hyperoxaluria type 1 (PH1), a rare autosomal recessive disorder characterized by excessive hepatic oxalate production due to a deficiency in alanine-glyoxylate aminotransferase (AGT) (Kukreja et al. (2019), Scott and Keam (2021)). This evidence concerns the gene AGXT and primary hyperoxaluria type 1.