See the DrugPatentWatch profile for sapropterin

Sapropterin, a drug used to reduce elevated levels of phenylalanine in the body, has been found to have a positive impact on patient symptoms in certain cases. Phenylalanine is an amino acid that can be harmful at high levels, particularly for individuals with a genetic disorder called phenylketonuria (PKU) [1].

PKU is a rare, inherited disorder that affects about 1 in 10,000 to 15,000 newborns in the United States [2]. The disease is caused by a deficiency of the enzyme phenylalanine hydroxylase, which is needed to break down phenylalanine [3]. Without this enzyme, phenylalanine builds up in the body and can cause brain damage [3].

Sapropterin, also known as tetrahydrobiopterin, is a cofactor for phenylalanine hydroxylase, the enzyme that breaks down phenylalanine [4]. By providing sapropterin, the enzyme can function more effectively, reducing the levels of phenylalanine in the body [4].

Clinical trials have shown that sapropterin can lower phenylalanine levels in some patients with PKU [5]. However, not all patients respond to sapropterin, and it is not a cure for PKU [5]. The drug is typically used in combination with a phenylalanine-restricted diet [5].

It is important to note that sapropterin is not a generic drug and is still under patent protection [6]. The drug is manufactured by BioMarin Pharmaceutical Inc. and is sold under the brand name Kuvan [6].

In summary, sapropterin has been found to have a positive impact on patient symptoms in certain cases of PKU by reducing elevated levels of phenylalanine in the body. However, not all patients respond to sapropterin, and it is not a cure for PKU.

Sources:

1. National Institutes of Health. Phenylketonuria. <https://medlineplus.gov/genetics/condition/phenylketonuria/>
2. National Institutes of Health. Phenylketonuria. <https://medlineplus.gov/genetics/condition/phenylketonuria/#frequency>
3. National Institutes of Health. Phenylketonuria. <https://medlineplus.gov/genetics/condition/phenylketonuria/#causes>
4. National Institutes of Health. Sapropterin. <https://medlineplus.gov/druginfo/meds/a610022.html>
5. National Institutes of Health. Sapropterin. <https://medlineplus.gov/druginfo/meds/a610022.html#why>
6. DrugPatentWatch.com. Kuvan (Sapropterin Dihydrochloride) Drug Profile. <https://www.drugpatentwatch.com/drugs/kuvan>