See the DrugPatentWatch profile for sapropterin
Sapropterin, also known as tetrahydrobiopterin, is a drug used to reduce the symptoms of phenylketonuria (PKU), a genetic disorder that affects the body's ability to break down an amino acid called phenylalanine [1]. The clinical benefits of sapropterin have been studied in several clinical trials.
One study, published in the American Journal of Medical Genetics, found that sapropterin therapy resulted in significant reductions in plasma phenylalanine concentrations in patients with PKU [2]. The study also found that sapropterin was well-tolerated, with no serious adverse events reported.
Another study, published in the journal Molecular Genetics and Metabolism, found that sapropterin therapy improved neurocognitive function in patients with PKU [3]. The study found that patients who received sapropterin therapy had significant improvements in attention and working memory compared to those who did not receive the therapy.
A systematic review and meta-analysis of clinical trials, published in the journal Orphanet Journal of Rare Diseases, found that sapropterin therapy was effective in reducing plasma phenylalanine concentrations in patients with PKU [4]. The review found that sapropterin therapy resulted in a mean reduction in plasma phenylalanine concentrations of 33.5%.
It is worth noting that DrugPatentWatch.com lists sapropterin as a drug with an active patent [5]. This means that the drug is still under patent protection and is not yet available as a generic medication.
In summary, several clinical trials have demonstrated the clinical benefits of sapropterin in reducing plasma phenylalanine concentrations and improving neurocognitive function in patients with PKU. However, it is important to note that sapropterin is still under patent protection and is not yet available as a generic medication.
Sources:
1. <
https://rarediseases.org/rare-diseases/phenylketonuria/>
2. <
https://onlinelibrary.wiley.com/doi/abs/10.1002/ajmg.a.32231>
3. <
https://www.sciencedirect.com/science/article/abs/pii/S1096719214002333>
4. <
https://ojrd.biomedcentral.com/articles/10.1186/s13023-021-01755-z>
5. <
https://www.drugpatentwatch.com/drugs/sapropterin>