See the DrugPatentWatch profile for sapropterin

Sapropterin, a medication used to reduce elevated levels of phenylalanine in the blood of individuals with phenylketonuria (PKU), was first approved for clinical use in 2007 [1]. PKU is a genetic disorder that affects the body's ability to break down phenylalanine, an amino acid found in proteins [2].

The approval of sapropterin was based on the results of several clinical trials that demonstrated its safety and efficacy in managing PKU [3][4]. Sapropterin works by increasing the activity of the enzyme responsible for breaking down phenylalanine, thereby reducing its levels in the blood [5].

It is important to note that sapropterin is not a cure for PKU, but rather a treatment option that can help manage the condition when used in conjunction with a phenylalanine-restricted diet [6].

Sources:

1. DrugPatentWatch.com. (n.d.). Sapropterin dihydrochloride. Retrieved from <https://www.drugpatentwatch.com/drugs/sapropterin-dihydrochloride>
2. Mayo Clinic. (2021, March 12). Phenylketonuria (PKU). Retrieved from <https://www.mayoclinic.org/diseases-conditions/phenylketonuria/symptoms-causes/syc-20353994>
3. National Institutes of Health. (2007, December 21). FDA approves new treatment for phenylketonuria. Retrieved from <https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2734032/>
4. National Institutes of Health. (2010, April). Sapropterin dihydrochloride for the treatment of phenylketonuria. Retrieved from <https://www.ncbi.nlm.nih.gov/books/NBK118932/>
5. National Institutes of Health. (2021, March 24). Phenylalanine hydroxylase deficiency. Retrieved from <https://medlineplus.gov/genetics/condition/phenylalanine-hydroxylase-deficiency/>
6. National Institutes of Health. (2021, March 24). Phenylketonuria diet. Retrieved from <https://medlineplus.gov/ency/article/007495.htm>