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.'
UroGen's UGN-103 IND accepted by FDA for bladder cancer treatment
MaaT Pharma reveals positive 18-month data for MaaT013 in GI-aGvHD
Innate Pharma advances Sanofi-developed NK cell engager to Phase 2 for blood cancer patients
Soligenix receives orphan drug designation from FDA for active ingredient in SuVax
Candel Therapeutics granted FDA Orphan Drug Designation for CAN-2409 in pancreatic cancer treatment
Lipogems completes patient enrolment in ARISE I US FDA IDE study
Amylyx Pharmaceuticals' AMX0035 shows promising impact on Wolfram syndrome symptoms
Roche attains CE Mark for first companion diagnostic for HER2-low metastatic breast cancer
Cadrenal Therapeutics' tecarfarin receives US FDA Orphan Drug Designation
BioCity Biopharma's BC2027 Phase one study IND application receives US FDA approval
Seres Therapeutics completes patient enrollment for SER-155 Phase 1B trial