See the DrugPatentWatch profile for sapropterin

Sapropterin is a synthetic form of the amino acid tetrahydrobiopterin (BH4), which plays a crucial role in the formation of coenzymes. Specifically, BH4 is a cofactor for the enzyme phenylalanine hydroxylase (PAH), which is responsible for converting phenylalanine to tyrosine in the body.

In the absence of BH4, PAH is unable to function properly, leading to the accumulation of phenylalanine and the development of phenylketonuria (PKU), a genetic disorder characterized by intellectual disability, seizures, and other neurological symptoms.

Sapropterin, as a synthetic form of BH4, is used to treat PKU by providing the necessary cofactor for PAH to function properly. By supplementing with sapropterin, individuals with PKU can convert phenylalanine to tyrosine, reducing the accumulation of toxic levels of phenylalanine in the body.

In addition to its role in treating PKU, sapropterin has also been shown to have potential therapeutic applications in other conditions, such as hyperphenylalaninemia and malignant melanoma.

Sources:

1. [1] National Institutes of Health. (2020). Phenylketonuria. Retrieved from <https://www.ncbi.nlm.nih.gov/books/NBK1365/>
2. [2] DrugPatentWatch.com. (2020). Sapropterin. Retrieved from <https://www.drugpatentwatch.com/drugs/sapropterin>
3. [3] PubMed. (2020). Tetrahydrobiopterin and its derivatives in the treatment of phenylketonuria. Retrieved from <https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7144449/>

Note: The sources cited above are a selection of the available information and are not exhaustive.