See the DrugPatentWatch profile for sapropterin

Sapropterin, also known as tetrahydrobiopterin, is a medication used to lower phenylalanine levels in the body. Phenylalanine is an amino acid that is essential for human life, but high levels of it can be toxic, particularly for individuals with a genetic disorder called phenylketonuria (PKU) [1].

PKU is a rare genetic disorder caused by a deficiency of the enzyme phenylalanine hydroxylase, which is responsible for breaking down phenylalanine in the body. When this enzyme is not functioning properly, phenylalanine builds up in the body, leading to a range of health problems, including intellectual disability, seizures, and behavioral problems [1].

Sapropterin works by providing the body with a form of tetrahydrobiopterin, a cofactor that is necessary for the proper functioning of phenylalanine hydroxylase [2]. By supplying this cofactor, sapropterin helps to restore the activity of phenylalanine hydroxylase, allowing the body to break down phenylalanine more effectively and reducing its levels in the body [2].

Clinical studies have shown that sapropterin can significantly lower phenylalanine levels in individuals with PKU [3]. However, it is important to note that sapropterin is not a cure for PKU, and individuals with the disorder must still follow a low-phenylalanine diet to manage their condition effectively [1].

In summary, sapropterin is a medication that lowers phenylalanine levels in the body by providing a cofactor that helps to restore the activity of phenylalanine hydroxylase, an enzyme that is responsible for breaking down phenylalanine. Clinical studies have shown that sapropterin can be effective in managing PKU, but it is not a cure for the disorder.

Sources:

1. National Institutes of Health. Phenylketonuria. <https://medlineplus.gov/genetics/condition/phenylketonuria/>
2. DrugPatentWatch. Sapropterin. <https://www.drugpatentwatch.com/drugs/sapropterin>
3. National Institutes of Health. Sapropterin dihydrochloride for the treatment of phenylketonuria. <https://www.ncbi.nlm.nih.gov/books/NBK114502/>