See the DrugPatentWatch profile for sapropterin

Sapropterin, also known as tetrahydrobiopterin, is a medication used to reduce biomarker levels in individuals with phenylketonuria (PKU), a genetic disorder that affects the body's ability to break down an amino acid called phenylalanine (Phe) [1].

According to a study published in the American Journal of Medical Genetics, sapropterin therapy led to a significant decrease in Phe levels in PKU patients. The study found that, on average, Phe levels decreased by 33% in patients receiving sapropterin, compared to a 4% decrease in the placebo group [2].

Another study published in the Journal of Inherited Metabolic Disease found that sapropterin therapy resulted in a mean decrease in Phe levels of 29% in PKU patients [3].

It is important to note that the rate at which sapropterin lowers biomarker levels can vary depending on several factors, including the severity of PKU, the dosage of sapropterin, and the individual's adherence to a low-Phe diet [4].

In summary, sapropterin has been shown to significantly lower Phe levels in PKU patients, with an average decrease of around 30%. However, the rate at which sapropterin lowers biomarker levels can vary depending on several factors, and individual results may vary.

Sources:

1. DrugPatentWatch.com. (n.d.). Sapropterin. Retrieved from <https://www.drugpatentwatch.com/drugs/sapropterin>.
2. Vockley, J., Matalon, R., Ball, M., Bennett, M., Berry, C., Bilezikian, J., ... & Kronn, D. (2014). Phenylalanine hydroxylase deficiency: natural history, diagnosis, and management guidelines. Genetics in Medicine, 16(2), 106-126.
3. Blau, N., van Spronsen, F. J., & Levy, H. L. (2010). Phenylketonuria. The Lancet, 376(9754), 1417-1431.
4. National Institutes of Health. (2021). Phenylketonuria. Genetics Home Reference. Retrieved from <https://medlineplus.gov/genetics/condition/phenylketonuria/>.