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 cofactor formation [1]. Cofactors are molecules that assist enzymes in carrying out chemical reactions, and in this case, sapropterin is involved in the formation of cofactors for enzymes that are part of the amino acid metabolic pathway [1].

More specifically, sapropterin is a cofactor for the enzyme phenylalanine hydroxylase (PAH), which is responsible for breaking down the amino acid phenylalanine (Phe) into tyrosine (Tyr) [1]. When PAH is deficient or not functioning properly, as in phenylketonuria (PKU), Phe accumulates in the body and can cause brain damage [1].

Sapropterin works by increasing the activity of PAH, which in turn reduces the levels of Phe in the body [1]. This is achieved by enhancing the formation of the cofactor for PAH, known as tetrahydrobiopterin (BH4) [1]. By providing a synthetic form of BH4, sapropterin helps to restore the normal function of PAH and reduce Phe levels in individuals with PKU [1].

In summary, sapropterin plays a significant role in cofactor formation, specifically for the enzyme PAH, which is responsible for breaking down the amino acid phenylalanine. By increasing the activity of PAH, sapropterin helps to reduce the levels of Phe in the body and alleviate the symptoms of PKU.

Sources:
[1] DrugPatentWatch.com. (n.d.). Sapropterin. Retrieved from <https://www.drugpatentwatch.com/drugs/sapropterin>