FDA grants Orphan Drug Designation to phenylketonuria treatment

A new treatment called APR-OD031 has been given Orphan Drug Designation (ODD) by the FDA, for the treatment of phenylketonuria. The US Food and Drug Administration (FDA) has announced that it has granted Orphan Drug Designation (ODD) to APR-OD031, for the treatment of phenylketonuria (PKU). The ODD was received by Applied Pharma Research (APR). APR-OD031 is an orally-delivered, extended-release amino acid mix engineered with a patented drug delivery technology enabling the secure physiological absorption of the delivered amino acids in the gut. According to APR, this is the first mix of amino acids having a pharmacological primary mode of action designed to reduce and control phenylalanine fluctuations and muscle proteolysis by reducing catabolic episodes in PKU patients, especially patients who do not respond to Sapropterin.

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