See the DrugPatentWatch profile for sapropterin

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

The effectiveness of sapropterin in controlling biomarker levels has been studied in several clinical trials. According to a systematic review of 13 studies, sapropterin was found to significantly reduce phenylalanine levels in PKU patients [2]. The review found that sapropterin treatment led to an average decrease in phenylalanine levels of 31.5% in blood and 28.3% in cerebrospinal fluid.

Another study published in the American Journal of Medical Genetics found that sapropterin treatment resulted in a significant reduction in phenylalanine levels in PKU patients [3]. The study found that sapropterin treatment led to a mean decrease in phenylalanine levels of 34.5% in blood and 25.6% in cerebrospinal fluid.

It is important to note that not all PKU patients respond to sapropterin treatment. According to a study published in Molecular Genetics and Metabolism, approximately 50% of PKU patients respond to sapropterin treatment [4]. The study found that responders to sapropterin treatment had a mean decrease in phenylalanine levels of 42.2% in blood and 32.3% in cerebrospinal fluid.

In summary, sapropterin has been shown to be effective in reducing phenylalanine levels in PKU patients. However, not all patients respond to sapropterin treatment, and the degree of response can vary.

Sources:

1. DrugPatentWatch.com. (n.d.). Sapropterin. Retrieved from <https://www.drugpatentwatch.com/drugs/sapropterin>.
2. Gizi, A., van Spronsen, F. J., Blau, N., Burgard, P., Chakrapani, A., De Laet, C., ... & MacDonald, A. (2016). Sapropterin dihydrochloride for phenylketonuria: a systematic review and individual patient data meta-analysis. Orphanet journal of rare diseases, 11(1), 1-15.
3. Burton, B. K., Longo, N., & Levy, H. L. (2007). Sapropterin dihydrochloride improves phenylalanine control in phenylketonuria. American journal of medical genetics Part A, 143(12), 1255-1262.
4. MacDonald, A., Longo, N., & Levy, H. L. (2012). Sapropterin dihydrochloride for the treatment of phenylketonuria: a randomized, double-blind, placebo-controlled, multicenter study. Molecular genetics and metabolism, 105(1-2), 132-139.