FDA grants Orphan Drug Designation to phenylketonuria treatment

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.

"The ODD for our drug candidate APR-OD031 is a huge achievement for a small company like APR, which is focusing its efforts on the improvement of PKU standard of care. Together with the other ODD granted six months ago, APR is strengthening its position as a patient-centric company dedicated to major unmet needs in the rare disease space," said Paolo Galfetti, Chief Executive Officer of APR. "We will work closely with the FDA to complete the development and bring to patients this new drug as soon as possible for the benefit of the PKU community still in the need of innovative and meaningful medications."

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