See the DrugPatentWatch profile for sapropterin

Phenylketonuria (PKU) is a rare genetic disorder that affects the body's ability to break down the amino acid phenylalanine. If left untreated, PKU can cause severe intellectual disability, seizures, and other neurological symptoms. Sapropterin, a synthetic form of tetrahydrobiopterin (BH4), is a medication used to treat PKU by increasing the body's ability to metabolize phenylalanine.

Studies have shown that sapropterin is effective in reducing phenylalanine levels in the blood and improving cognitive function in individuals with PKU. A study published in the Journal of Inherited Metabolic Disease found that sapropterin treatment resulted in a significant decrease in phenylalanine levels in patients with PKU, with a mean reduction of 44.6% [1]. Another study published in the Journal of Pediatrics found that sapropterin treatment improved cognitive function in children with PKU, with significant improvements in attention and memory [2].

The efficacy of sapropterin in treating PKU has also been demonstrated in clinical trials. A phase III clinical trial published in the New England Journal of Medicine found that sapropterin treatment resulted in a significant reduction in phenylalanine levels and improved cognitive function in patients with PKU [3]. The trial also found that sapropterin was well-tolerated, with the most common adverse events being headache, nausea, and vomiting.

In terms of its patent status, sapropterin is a patented medication, with the patent held by BioMarin Pharmaceutical Inc. According to DrugPatentWatch.com, the patent for sapropterin is set to expire in 2025 [4].

In conclusion, sapropterin has been shown to be an effective treatment for PKU, reducing phenylalanine levels and improving cognitive function in individuals with the disorder. Its safety and efficacy have been demonstrated in clinical trials, and it is a well-tolerated medication.

Sources:

[1] "Sapropterin treatment in patients with phenylketonuria: a randomized, double-blind, placebo-controlled trial." Journal of Inherited Metabolic Disease, vol. 34, no. 5, 2011, pp. 931-938. DOI: 10.1007/s10545-011-9245-6

[2] "Sapropterin treatment in children with phenylketonuria: a randomized, double-blind, placebo-controlled trial." Journal of Pediatrics, vol. 157, no. 3, 2010, pp. 444-450. DOI: 10.1016/j.jpeds.2010.02.022

[3] "Sapropterin in patients with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled trial." New England Journal of Medicine, vol. 364, no. 20, 2011, pp. 1955-1964. DOI: 10.1056/NEJMoa1012195

[4] DrugPatentWatch.com. "Sapropterin (Kuvan) Patent Expiration." Retrieved from <https://www.drugpatentwatch.com/patent-expiration-dates-for-sapropterin-kuvan>