The United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to Switzerland-based APR Applied Pharma Research SA's drug candidate, APR-OD031, intended for the treatment of Phenylketonuria (PKU), an inherited, recessive, metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase, it was reported on Thursday.
The product is an extended release amino acid mix designed with a patented drug delivery technology that enables the securing of physiological absorption of the delivered amino acids.
Paolo Galfetti, CEO of APR Applied Pharma Research SA, said, '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. As father of a PKU boy and as professional in this industry, I feel the privilege and the honour 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.'
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