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Long-term efficacy and safety of sapropterin in patients who initiated sapropterin at < 4 years of age with phenylketonuria: results of the 3-year extension of the SPARK open-label, multicentre, randomised phase IIIb trial

During the initial 26-week SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) study, addition of sapropterin dihydrochloride (Kuvan®; a synthetic formulation of the natural cofactor for phenylalanine hydroxylase, tetrahydrobiopterin; BH4), to a phenylalanine (Phe)-restricted diet, led to...

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Main Authors: Muntau, Ania (Author) , Burlina, Alberto (Author) , Eyskens, François (Author) , Freisinger, Peter (Author) , Leuzzi, Vincenzo (Author) , Sivri, Hatice Serap (Author) , Gramer, Gwendolyn (Author) , Pazdírková, Renata (Author) , Cleary, Maureen (Author) , Lotz-Havla, Amelia S. (Author) , Lane, Paul (Author) , Alvarez, Ignacio (Author) , Rutsch, Frank (Author)
Format: Article (Journal)
Language:English
Published: 03 August 2021
In: Orphanet journal of rare diseases
Year: 2021, Volume: 16, Pages: 1-11
ISSN:1750-1172
DOI:10.1186/s13023-021-01968-1
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1186/s13023-021-01968-1
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Author Notes:Ania C. Muntau, Alberto Burlina, François Eyskens, Peter Freisinger, Vincenzo Leuzzi, Hatice Serap Sivri, Gwendolyn Gramer, Renata Pazdírková, Maureen Cleary, Amelia S. Lotz-Havla, Paul Lane, Ignacio Alvarez and Frank Rutsch
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Summary:During the initial 26-week SPARK (Safety Paediatric efficAcy phaRmacokinetic with Kuvan®) study, addition of sapropterin dihydrochloride (Kuvan®; a synthetic formulation of the natural cofactor for phenylalanine hydroxylase, tetrahydrobiopterin; BH4), to a phenylalanine (Phe)-restricted diet, led to a significant improvement in Phe tolerance versus a Phe-restricted diet alone in patients aged 0-4 years with BH4-responsive phenylketonuria (PKU) or mild hyperphenylalaninaemia (HPA). Based on these results, the approved indication for sapropterin in Europe was expanded to include patients < 4 years of age. Herein, we present results of the SPARK extension study (NCT01376908), evaluating the long-term safety, dietary Phe tolerance, blood Phe concentrations and neurodevelopmental outcomes in patients < 4 years of age at randomisation, over an additional 36 months of treatment with sapropterin.
Item Description:Gesehen am 25.10.2021
Physical Description:Online Resource
ISSN:1750-1172
DOI:10.1186/s13023-021-01968-1

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