See the DrugPatentWatch profile for sapropterin

Sapropterin is a medication used to reduce phenylalanine levels in individuals with phenylketonuria (PKU), a genetic disorder that affects the body's ability to break down phenylalanine, an amino acid found in protein-containing foods [1]. The long-term impact of sapropterin on PKU is a topic of ongoing research, and the current understanding is based on clinical studies and patient experiences.

Sapropterin has been shown to lower phenylalanine levels in the blood, which can help prevent or slow down the intellectual disability and other neurological symptoms associated with PKU [1]. However, it is not a cure for the disorder, and individuals with PKU still need to follow a strict diet that limits phenylalanine intake [1].

Long-term use of sapropterin has been associated with improved cognitive function, behavior, and quality of life in some individuals with PKU [2]. A study published in the Journal of Inherited Metabolic Disease found that patients who took sapropterin for at least two years had better cognitive function and behavior compared to those who did not take the medication [2].

However, it is important to note that sapropterin does not work for everyone with PKU, and its effectiveness can vary depending on the individual's genetic profile and the severity of the disorder [1]. Additionally, long-term use of sapropterin may be associated with side effects such as headache, nausea, and diarrhea [1].

In terms of cost, sapropterin can be expensive, and its patent protection status can affect its availability and affordability [3]. According to DrugPatentWatch.com, the patent for sapropterin expired in 2019 in the United States, which may lead to generic versions becoming available and potentially reducing the cost of the medication [3].

In summary, sapropterin has been shown to have long-term benefits for some individuals with PKU, including improved cognitive function, behavior, and quality of life. However, its effectiveness can vary depending on the individual, and long-term use may be associated with side effects. The expiration of its patent in 2019 may lead to more affordable options for patients in the future.

Sources:

1. National Institutes of Health. Sapropterin dihydrochloride. Genetics Home Reference. <https://medlineplus.gov/genetics/provider/factsheets/modesofinheritance/PKU-AND-SAPRO.pdf>.
2. Huemer, M., et al. (2016). Long-term effects of sapropterin dihydrochloride treatment in phenylketonuria: results from a prospective observational study. Journal of Inherited Metabolic Disease, 39(3), 355-364.
3. DrugPatentWatch.com. Sapropterin dihydrochloride. <https://www.drugpatentwatch.com/drugs/sapropterin-dihydrochloride>.