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FDA Grants Orphan Drug Designation to APR-OD031 for Phenylketonuria

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Applied Pharma Research s.a. (APR), the Swiss pharma company leveraging drug delivery technologies to develop innovative products for niche and rare diseases, today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for the Company’s drug candidate APR-OD031 for the treatment of Phenylketonuria (“PKU”).

APR-OD031 is an extended release amino acid mix engineered with a patented drug delivery technology enabling to secure physiological absorption of the delivered amino acids. This is the first mix of amino acids having a pharmacological primary mode of action designed to reduce and control Phenylalanine (“Phe”) fluctuations and muscle proteolysis by reducing catabolic episodes in PKU Patients, especially Classic PKU Patients not responding to Sapropterin.

“The Orphan Drug Designation 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 orphan drug designation granted 6 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, CEO of APR in a statement. “As father of a PKU boy and as professional in this industry, I feel the privilege and the honor for leading a group of passionate people, all sharing the same vision to improve the quality of life of patients and families living with rare diseases. We will work closely with the FDA to complete the development and bring to the Patients this new drug as soon as possible for the benefit of the PKU community still in the need of innovative and meaningful medications.”

Source: Applied Pharma Research s.a

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