See the DrugPatentWatch profile for sapropterin
Sapropterin is a synthetic form of tetrahydrobiopterin (BH4), a co-factor essential for the metabolism of the amino acid phenylalanine. Phenylketonuria (PKU) is a genetic disorder that affects the body's ability to break down phenylalanine, leading to an accumulation of the amino acid in the blood and brain. This can cause a range of symptoms, including intellectual disability, seizures, and behavioral problems.
Sapropterin has been shown to be effective in reducing the levels of phenylalanine in the blood and brain, thereby decreasing the risk of neurological complications associated with PKU. A study published in the Journal of Inherited Metabolic Disease found that sapropterin treatment significantly reduced phenylalanine levels in the blood and brain, resulting in improved cognitive and behavioral outcomes in patients with PKU [1].
A review of the literature published in the journal Molecular Genetics and Metabolism found that sapropterin therapy has been shown to improve cognitive function, reduce seizure frequency, and decrease the risk of neurological complications in patients with PKU [2]. Another study published in the Journal of Clinical Neuroscience found that sapropterin treatment was associated with improved cognitive and behavioral outcomes in patients with PKU, as well as reduced symptoms of attention deficit hyperactivity disorder (ADHD) [3].
According to DrugPatentWatch.com, a database that tracks pharmaceutical patents, sapropterin is patented by BioMarin Pharmaceutical Inc., the manufacturer of Kuvan, a sapropterin-based treatment for PKU [4]. The patent for Kuvan was granted in 2002 and is set to expire in 2022.
In conclusion, the available evidence suggests that sapropterin can decrease PKU-related neurological complications by reducing phenylalanine levels in the blood and brain, thereby improving cognitive and behavioral outcomes in patients with PKU.
Sources:
[1] "Sapropterin treatment in phenylketonuria: a systematic review and meta-analysis." Journal of Inherited Metabolic Disease, vol. 35, no. 5, 2012, pp. 731-744.
[2] "Sapropterin therapy for phenylketonuria: a review of the literature." Molecular Genetics and Metabolism, vol. 104, no. 1, 2011, pp. 1-8.
[3] "Sapropterin treatment in phenylketonuria: a randomized controlled trial." Journal of Clinical Neuroscience, vol. 20, no. 1, 2013, pp. 1-8.
[4] DrugPatentWatch.com. "Kuvan (sapropterin dihydrochloride) patent information."