FDA Grants Orphan Drug Designation for Phenylketonuria Treatment

The US Food and Drug Administration (FDA) has granted orphan drug status to APR-OD031, an extended release amino acid mix to treat phenylketonuria (PKU).

Applied Pharma Research (APR), a pharmaceutical company based in Switzerland, was given the status for the drug, which was engineered with a patented drug delivery technology that enables the secure physiological absorption of the delivered amino acids.

The treatment represents the first mix of amino acids having a pharmacological primary mode of action that reduces and controls phenylalanine (Phe) fluctuations and muscle proteolysis by reducing catabolic episodes common in PKU patients, particularly classic PKU patients that do not respond to sapropterin.

The new drug is aimed at modifying the release of Phe-free amino acids with an absorption profile that mimics dietary proteins.

When used in conjunction with the Physiomimic Technology drug delivery system, APR-OD031 enables the production of small coated granules for oral administration, processed to gradually release the amino acids in the patient’s gut.

"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,” Paolo Galfetti, CEO of APR, said in a statement. “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."