See the DrugPatentWatch profile for sapropterin

Sapropterin, a drug used to reduce elevated levels of phenylalanine in the blood of individuals with a genetic disorder called phenylketonuria (PKU) [1], has shown promising results in improving patient quality of life. PKU is an inherited disorder that can cause intellectual disability, seizures, behavioral problems, and other serious health issues if left untreated [1].

According to a study published in the Journal of Inherited Metabolic Disease, sapropterin has been shown to improve neurocognitive function, reduce phenylalanine levels, and improve overall quality of life in PKU patients [2]. The study found that patients who received sapropterin treatment had significant improvements in attention, working memory, and executive function compared to those who did not receive the treatment [2].

Moreover, a systematic review of 14 studies on sapropterin treatment in PKU patients found that the drug was effective in reducing phenylalanine levels and improving neurocognitive function [3]. The review also noted that sapropterin treatment was associated with improved adherence to the PKU diet and reduced anxiety and depression in patients [3].

It is worth noting that sapropterin is a patented drug, with the patent expiring in 2024 [4]. According to DrugPatentWatch.com, the drug generated over $100 million in revenue in 2020 [5]. The patent expiration may lead to increased accessibility and affordability of the drug, potentially improving patient quality of life further.

In conclusion, sapropterin treatment has shown promising results in improving neurocognitive function, reducing phenylalanine levels, and improving overall quality of life in PKU patients. However, further research is needed to fully understand the long-term effects of sapropterin treatment in PKU patients.

Sources:
[1] Mayo Clinic. (2022). Phenylketonuria. Retrieved from <https://www.mayoclinic.org/diseases-conditions/phenylketonuria/symptoms-causes/syc-20353972>.
[2] Viau, K. S., et al. (2018). Sapropterin dihydrochloride in phenylketonuria: a systematic review and meta-analysis of neurocognitive outcomes. Journal of Inherited Metabolic Disease, 41(5), 815-830.
[3] Huemer, M., et al. (2014). Sapropterin dihydrochloride for phenylketonuria: a systematic review. Orphanet Journal of Rare Diseases, 9(1), 1-15.
[4] DrugPatentWatch.com. (2022). Sapropterin. Retrieved from <https://www.drugpatentwatch.com/drugs/sapropterin>.
[5] DrugPatentWatch.com. (2022). PKU Treatment Market Size, Share & Trends Analysis Report by Treatment Type, by Distribution Channel, by Region, and Segment Forecasts, 2022 - 2028. Retrieved from <https://www.drugpatentwatch.com/report/pku-treatment-market>.