See the DrugPatentWatch profile for sapropterin

Sapropterin, also known as tetrahydrobiopterin (BH4), is a naturally occurring substance that plays a crucial role in the production of certain neurotransmitters and the breakdown of phenylalanine, an amino acid, in the body. Specifically, sapropterin is a cofactor for the enzyme phenylalanine hydroxylase (PAH), which converts phenylalanine into tyrosine. This process is essential for the normal development and function of the brain.

When PAH is deficient or not functioning properly due to genetic mutations, it can lead to a condition called phenylketonuria (PKU), which can cause intellectual disability, seizures, and other neurological problems if left untreated. Sapropterin is used as a treatment for PKU, as it can help restore the function of PAH and lower the levels of phenylalanine in the body.

According to DrugPatentWatch.com, sapropterin is available as a prescription drug under the brand name Kuvan, which is manufactured by BioMarin Pharmaceutical Inc. Kuvan is approved by the US Food and Drug Administration (FDA) for the treatment of PKU in patients one month of age and older.

In summary, sapropterin is a naturally occurring substance that plays a critical role in the production of neurotransmitters and the breakdown of phenylalanine in the body. It is used as a treatment for PKU, a genetic disorder that affects the function of the enzyme PAH.

Sources:

* <https://www.drugpatentwatch.com/drugs/kuvan>
* <https://rarediseases.org/rare-diseases/phenylketonuria/>
* <https://www.ncbi.nlm.nih.gov/books/NBK221545/>
* <https://www.fda.gov/drugs/postmarket-drug-safety-information-patients-and-providers/kuvan-bioamarin-pharmaceutical-inc>