FDA Grants Orphan Drug Designation to APR-OD031 for the Treatment of Phenylketonuria (PKU)

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FDA Grants Orphan Drug Designation to APR-OD031 for the Treatment of Phenylketonuria (PKU)

26.03.2020 - 16:15

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BALERNA, Switzerland, March 26, 2020 /PRNewswire/ -- APR 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 (the "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. "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."

About APR-OD031

APR-OD031 is an extended-release, Phe-free, amino acids containing formula designed to modify their release and absorption profile to mimic that of dietary proteins. APR-OD031 is engineered using the patented drug delivery Physiomimic Technology™ enabling the production of small coated granules for oral administration, processed to gradually release the amino acids in the gut. APR-OD031 is intended to reduce Phenylalanine fluctuations and muscle proteolysis in PKU patients not responding to Sapropterin. Moreover, the Physiomimic Technology™ is designed to improve patient compliance and adherence to treatments because it remarkably masks the taste and the odour of Amino Acids, with positive consequences on the typical unpleasant taste, odour, and aftertaste of traditional formulations. 

About Phenylketonuria (PKU)

PKU (Phenylketonuria) is an inherited, recessive, metabolic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase ("PAH"). In most of the countries worldwide, PKU occurs in 1 in 10,000 to 1 in 15,000 new born babies, according to the National Human Genome Research Institute. This enzyme is required for the breakdown of Phenylalanine ("Phe"), an amino acid found in all protein-containing foods. When PAH is deficient or defective, Phe accumulates to abnormally high levels in the blood and consequently into the brain. Clinical manifestations of sustained high Phe levels include a variety of serious neurological complications, including intellectual disability and brain damage, mood disturbances, seizures and tremors, cognitive problems and executive function deficits which are clinically relevant not only in children and young people but also in adults.

About APR Applied Pharma Research s.a.

APR is a Swiss pharma company focused on development and commercialization of innovative, research-driven products designed to address unmet needs in niche and rare therapeutic areas. APR combines pharmaceutical development expertise with proprietary drug delivery technologies to develop solutions that meaningfully improve the lives of people with rare diseases. A diverse and balanced portfolio of revenue-generating products in all major markets is complemented by a robust pipeline of innovative products at different stages of development for the treatment of recessive metabolic disorders, as well as rare dermatological and ocular diseases. Products are commercialized by APR with its sales and marketing teams in selected countries of Europe and by a growing network of commercial partners. For more information, please visit: https://www.apr.ch.

Forward-looking statements

The contents of this announcement include statements that are, or may be deemed to be, "forward-looking statements". These forward-looking statements can be identified by the use of forward-looking terminology, including the words "believes", "estimates," "expects", "intends", "may", "will", "plans", "continue", "ongoing", "potential", "project", "seek" or "should", and include statements the Company makes concerning the intended results of its strategy. By their nature, forward-looking statements involve risks and uncertainties and readers are cautioned that any such forward-looking statements are not guarantees of future performance. The Company's actual results may differ materially from those predicted by the forward-looking statements. The Company undertakes no obligation to publicly update or revise forward-looking statements, except as may be required by law.

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Contact: APR Applied Pharma Research s.a.:Paolo Galfetti, Chief Executive Officerpaolo.galfetti@apr.ch +41-91-6957020

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