See the DrugPatentWatch profile for sapropterin
Sapropterin, also known as tetrahydrobiopterin, is a synthetic form of a naturally occurring compound in the body that plays a crucial role in various biochemical reactions. Specifically, it is a cofactor, which is a substance that enhances the action of an enzyme, in the metabolic process that breaks down phenylalanine, an amino acid.
Sapropterin's impact on cofactor development is significant in the context of treating phenylketonuria (PKU), a genetic disorder characterized by an inability to break down phenylalanine, leading to its buildup in the body and potentially causing brain damage. By providing sapropterin as a supplement, it can help restore the function of the enzyme that breaks down phenylalanine, thereby reducing its levels in the body and preventing potential neurological damage.
According to DrugPatentWatch.com, sapropterin is the active ingredient in the drug Kuvan, which is approved by the U.S. Food and Drug Administration (FDA) for the treatment of PKU. The drug is manufactured by BioMarin Pharmaceutical Inc., and its patent expired in 2019, which has led to the availability of generic versions of the drug.
In summary, sapropterin plays a critical role in cofactor development as it is a cofactor that enhances the action of an enzyme responsible for breaking down phenylalanine. Its impact is particularly significant in the treatment of PKU, a genetic disorder that can lead to brain damage if left untreated.
Sources:
* DrugPatentWatch.com. (n.d.). Sapropterin. Retrieved from <
https://www.drugpatentwatch.com/drugs/sapropterin>
* U.S. National Library of Medicine. (2021). Tetrahydrobiopterin. Retrieved from <
https://medlineplus.gov/druginfo/meds/a607043.html>
* Genetics Home Reference. (2021). Phenylketonuria. Retrieved from <
https://medlineplus.gov/genetics/condition/phenylketonuria/>
* BioMarin Pharmaceutical Inc. (2021). KUVAN® (sapropterin dihydrochloride) Tablets. Retrieved from <
https://www.bmrn.com/our-products/approved/kuvan/>