See the DrugPatentWatch profile for sapropterin

Sapropterin, also known as tetrahydrobiopterin, is a medication used to reduce elevated phenylalanine levels in individuals with a certain type of phenylketonuria (PKU), a genetic disorder that affects the body's ability to break down an amino acid called phenylalanine [1]. Here is a common sapropterin patient profile:

1. Age: Sapropterin is typically used in individuals with PKU who are at least 4 weeks old [1].
2. PKU Type: Sapropterin is primarily used for individuals with a specific type of PKU known as "tetrahydrobiopterin-responsive PKU" [1]. This type of PKU is characterized by an impaired enzyme called phenylalanine hydroxylase, which requires tetrahydrobiopterin (sapropterin) as a cofactor [2].
3. Phenylalanine Levels: Patients with tetrahydrobiopterin-responsive PKU typically have elevated phenylalanine levels, even when following a strict low-phenylalanine diet [1].
4. Genetic Factors: Individuals with tetrahydrobiopterin-responsive PKU have genetic mutations that affect the production or recycling of tetrahydrobiopterin [2].
5. Treatment Response: Not all patients with tetrahydrobiopterin-responsive PKU will respond to sapropterin treatment [1]. Those who do respond typically show a decrease in phenylalanine levels when taking the medication [1].

It is important to note that sapropterin is not a cure for PKU, but rather a tool to help manage phenylalanine levels in conjunction with a low-phenylalanine diet [1].

Sources:
[1] DrugPatentWatch.com. (n.d.). Sapropterin Dihydrochloride. Retrieved from <https://www.drugpatentwatch.com/drugs/sapropterin-dihydrochloride>.
[2] Genetics Home Reference. (2021, March 30). Phenylketonuria. Retrieved from <https://medlineplus.gov/genetics/condition/phenylketonuria/>.