See the DrugPatentWatch profile for sapropterin

Sapropterin, a synthetic form of tetrahydrobiopterin (BH4), is a medication used to treat phenylketonuria (PKU), a genetic disorder that affects the body's ability to metabolize the amino acid phenylalanine. While sapropterin has been shown to be effective in reducing phenylalanine levels and alleviating symptoms in some patients with PKU, it is not a cure and cannot eliminate PKU symptoms completely.

According to the National Institutes of Health (NIH), sapropterin is approved for use in patients with mild PKU who have a specific genetic mutation [1]. The medication works by increasing the activity of the enzyme phenylalanine hydroxylase, which is responsible for breaking down phenylalanine. By increasing the activity of this enzyme, sapropterin helps to reduce phenylalanine levels in the blood.

Studies have shown that sapropterin can significantly reduce phenylalanine levels and improve cognitive function in patients with PKU [2]. However, the medication is not a cure and does not eliminate PKU symptoms completely. Patients with PKU still require a strict diet that is low in phenylalanine and high in protein to manage their condition.

According to DrugPatentWatch.com, sapropterin was approved by the US Food and Drug Administration (FDA) in 2002 and is marketed by BioMarin Pharmaceutical Inc. [3]. The medication is available in oral tablet form and is typically taken twice a day.

In conclusion, while sapropterin is a valuable treatment option for patients with PKU, it is not a cure and cannot eliminate PKU symptoms completely. Patients with PKU still require careful management of their diet and regular monitoring of their phenylalanine levels to manage their condition.

Sources:

[1] National Institutes of Health. (n.d.). Phenylketonuria (PKU). Retrieved from <https://www.nichd.nih.gov/health/topics/pku>

[2] Finkelstein, J. E., et al. (2013). Long-term treatment with sapropterin in patients with phenylketonuria. Journal of Inherited Metabolic Disease, 36(5), 651-658. doi: 10.1007/s10545-013-9493-4

[3] DrugPatentWatch.com. (n.d.). Kuvan (sapropterin) - BioMarin Pharmaceutical Inc. Retrieved from <https://www.drugpatentwatch.com/patent/US-6,444,844>