APR-OD032

APR-OD032 (also known as RLF-OD032) is a concentrated, ready-to-use liquid oral formulation of sapropterin dihydrochloride, a synthetic analog of the naturally occurring cofactor tetrahydrobiopterin (BH4). Developed by APR Applied Pharma Research (a subsidiary of Relief Therapeutics), it is intended for the treatment of phenylketonuria (PKU), a rare genetic disorder characterized by a deficiency in the enzyme phenylalanine hydroxylase (PAH). By providing exogenous BH4, the drug enhances the activity of residual PAH, facilitating the metabolism of phenylalanine into tyrosine and reducing toxic blood phenylalanine levels. APR-OD032 is designed to improve patient compliance and quality of life compared to existing tablet and powder formulations through its portable, flavored, and ready-to-administer format. The product is being developed via the FDA's 505(b)(2) regulatory pathway, utilizing bioequivalence data to the reference listed drug, Kuvan.

Other names

sapropterin dihydrochloride liquid formulation