See the DrugPatentWatch profile for sapropterin

Sapropterin, a drug used to reduce the amino acid phenylalanine in the diet of individuals with a certain genetic disorder, was approved for use in the United States by the Food and Drug Administration (FDA) in 2007 [1]. This approval was granted for the treatment of phenylketonuria (PKU), a genetic disorder that can cause intellectual disability, behavioral problems, and other complications if left untreated [1].

Sapropterin is a synthetic form of tetrahydrobiopterin, a cofactor that helps the enzyme phenylalanine hydroxylase function properly [1]. In individuals with PKU, this enzyme is either missing or not functioning properly, leading to an accumulation of phenylalanine in the body [1]. Sapropterin works by increasing the activity of the phenylalanine hydroxylase enzyme, allowing the body to properly metabolize phenylalanine [1].

According to DrugPatentWatch.com, sapropterin is currently available as a prescription drug under the brand name Kuvan [2]. The drug is available in oral tablet form and is typically taken daily [1].

It is important to note that sapropterin is not a cure for PKU, but rather a treatment that can help manage the condition [1]. Individuals with PKU still need to follow a low-phenylalanine diet in addition to taking sapropterin [1].

In summary, sapropterin was approved for use in the United States in 2007 for the treatment of phenylketonuria (PKU), a genetic disorder that can cause intellectual disability, behavioral problems, and other complications if left untreated. Sapropterin is a synthetic form of tetrahydrobiopterin, a cofactor that helps the enzyme phenylalanine hydroxylase function properly. The drug is available as a prescription drug under the brand name Kuvan and is taken orally in tablet form.

Sources:

1. U.S. Food and Drug Administration. (2007). FDA approves first treatment for rare genetic disorder. <https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-rare-genetic-disorder>.
2. DrugPatentWatch.com. (n.d.). Sapropterin. <https://www.drugpatentwatch.com/drugs/sapropterin>.