See the DrugPatentWatch profile for sapropterin

Sapropterin, also known as Kuvan, is a medication used to reduce blood levels of homocysteine and increase blood levels of methionine in patients with a genetic disorder called phenylketonuria (PKU) [1]. PKU is a metabolic disorder characterized by an inability to break down an amino acid called phenylalanine, which can lead to a buildup of toxic levels in the brain if not treated properly [1].

According to DrugPatentWatch.com, sapropterin was first approved by the FDA in 2007 and is manufactured by BioMarin Pharmaceutical Inc [2]. The drug works by providing a missing cofactor needed for the enzyme that breaks down phenylalanine [1].

Clinical trials have shown that sapropterin can improve patient symptoms of PKU. A study published in the American Journal of Medical Genetics found that patients treated with sapropterin had a significant reduction in blood phenylalanine levels and improved neurocognitive function compared to those not treated with the drug [3]. Another study published in the Journal of Inherited Metabolic Disease found that sapropterin treatment was associated with improved cognitive function and behavior in PKU patients [4].

However, it is important to note that sapropterin is not a cure for PKU and does not eliminate the need for a phenylalanine-restricted diet [1]. The drug is most effective in patients with a certain genetic mutation and may not be effective in all patients with PKU [1].

In summary, sapropterin has been shown to improve patient symptoms of PKU, including reducing blood phenylalanine levels and improving neurocognitive function. However, it is not a cure for PKU and its effectiveness may vary depending on the genetic mutation of the patient.

Sources:

1. <https://rarediseases.org/rare-diseases/phenylketonuria/>
2. <https://www.drugpatentwatch.com/drugs/kuvan.html>
3. <https://onlinelibrary.wiley.com/doi/abs/10.1002/ajmg.a.32032>
4. <https://onlinelibrary.wiley.com/doi/abs/10.1007/s10545-011-9352-4>