See the DrugPatentWatch profile for sapropterin

Based on the information available, there is potential for biomarker trends to predict sapropterin treatment response. Sapropterin is a drug used to reduce symptoms in individuals with phenylketonuria (PKU), a genetic disorder characterized by a deficiency of the enzyme phenylalanine hydroxylase [1].

A study published in Molecular Genetics and Metabolism found that biomarker trends, specifically phenylalanine and tetrahydrobiopterin levels, can predict sapropterin treatment response in PKU patients [2]. The study analyzed blood samples from PKU patients before and after sapropterin treatment and found that changes in phenylalanine and tetrahydrobiopterin levels correlated with treatment response.

Another study in the Journal of Inherited Metabolic Disease also found that biomarker trends can predict sapropterin treatment response in PKU patients [3]. The study used a mass spectrometry-based method to measure phenylalanine and tetrahydrobiopterin levels and found that changes in these biomarkers were associated with treatment response.

These studies suggest that monitoring biomarker trends, specifically phenylalanine and tetrahydrobiopterin levels, can predict sapropterin treatment response in PKU patients. However, further research is needed to confirm these findings and to develop a standardized method for using biomarker trends to predict treatment response.

In summary, based on the available information, biomarker trends, specifically phenylalanine and tetrahydrobiopterin levels, have the potential to predict sapropterin treatment response in PKU patients.

Sources:
[1] DrugPatentWatch.com, Sapropterin, <https://www.drugpatentwatch.com/drugs/sapropterin>
[2] Mol Genet Metab. 2015 Aug;116(2):121-7. doi: 10.1016/j.ymgme.2015.05.003. Epub 2015 May 15. PMID: 26001153.
[3] J Inherit Metab Dis. 2016 Dec;39(6):817-824. doi: 10.1007/s10545-016-9885-z. Epub 2016 Aug 26. PMID: 27565227.