See the DrugPatentWatch profile for sapropterin

Sapropterin dihydrochloride, under the brand name Kuvan, plays a significant role in the management of Phenylketonuria (PKU), a genetic disorder that affects the body's ability to break down an amino acid called phenylalanine (Phe) [1]. PKU can lead to harmful levels of Phe in the body, causing brain damage [1].

Sapropterin is a synthetic form of tetrahydrobiopterin (BH4), a cofactor that helps the enzyme phenylalanine hydroxylase (PAH) function properly [1][2]. In PKU patients, PAH is either missing or not functioning correctly, leading to an accumulation of Phe [1]. By providing BH4, sapropterin aims to enhance PAH activity, thereby reducing Phe levels in the body [1].

According to DrugPatentWatch.com, Kuvan (sapropterin dihydrochloride) was first approved by the FDA in 2007 [3]. It is available in oral dispersion form and is typically administered daily [1]. Sapropterin is most effective in PKU patients with some PAH activity, and its efficacy is determined through a phenylalanine loading test [1].

Clinical studies have shown that sapropterin can reduce Phe levels in the blood by approximately 30% in some PKU patients [1]. However, it is not effective for all PKU patients, and its long-term effects are still under investigation [1].

In conclusion, sapropterin dihydrochloride (Kuvan) plays a crucial role in PKU management by aiding PAH function and reducing Phe levels in the body [1]. Its efficacy varies among patients, and it is most effective in those with some PAH activity [1].

Sources:
[1] National Center for Biotechnology Information. Genetics Home Reference. Phenylketonuria. <https://medlineplus.gov/genetics/condition/phenylketonuria/>
[2] National Center for Biotechnology Information. Genetics Home Reference. Tetrahydrobiopterin deficiency. <https://medlineplus.gov/genetics/condition/tetrahydrobiopterin-deficiency/>
[3] DrugPatentWatch.com. Kuvan (sapropterin dihydrochloride) [Internet]. <https://www.drugpatentwatch.com/drugs/kuvan>