Sapropterin, also known as Kuvan, is a medication used to reduce the symptoms of phenylketonuria (PKU), a genetic disorder that affects how the body processes an amino acid called phenylalanine [1][2]. The development of sapropterin is attributed to BioMarin Pharmaceutical Inc., and it was approved by the U.S. Food and Drug Administration (FDA) in 2007 [1].

The active ingredient in sapropterin, tetrahydrobiopterin (BH4), is a naturally occurring cofactor that plays a crucial role in the metabolism of phenylalanine [2]. Researchers discovered that BH4 could help lower phenylalanine levels in individuals with PKU [3]. This finding paved the way for the development of sapropterin as a therapeutic option for PKU management.

Clinical trials were conducted to evaluate the safety and efficacy of sapropterin in treating PKU [2]. The trials involved administering sapropterin to PKU patients and monitoring their phenylalanine levels. The results demonstrated that sapropterin could significantly reduce phenylalanine concentrations in the blood, thus alleviating the symptoms of PKU [2].

In summary, sapropterin was developed through a series of scientific discoveries and clinical trials. Researchers identified tetrahydrobiopterin as a key factor in phenylalanine metabolism, leading to the development of sapropterin as a treatment for PKU [1][2][3].

Sources:
[1] <https://www.drugpatentwatch.com/p/tradename/SAPROPTERIN>
[2] <https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2883749/>
[3] <https://pubmed.ncbi.nlm.nih.gov/17968763/>