See the DrugPatentWatch profile for sapropterin

Sapropterin, also known as (6R)-L-erythro-5,6,7,8-tetrahydrobiopterin, is a drug used to reduce the symptoms of Phenylketonuria (PKU) [1]. PKU is a genetic disorder characterized by the body's inability to break down an amino acid called phenylalanine [2]. If left untreated, PKU can lead to intellectual disability, seizures, behavioral problems, and other serious health issues [2].

Sapropterin is a cofactor for the phenylalanine hydroxylase (PAH) enzyme, which is responsible for breaking down phenylalanine [1]. By providing a sufficient amount of sapropterin, PAH function can be restored, thereby reducing the levels of phenylalanine in the body [1].

According to DrugPatentWatch.com, sapropterin is indicated for the treatment of PKU in patients who have a certain type of PAH deficiency [1]. The drug can help to reduce phenylalanine levels in the blood, which can alleviate some of the symptoms associated with PKU [1].

Some of the specific symptoms of PKU that may be eased by sapropterin include:

1. Neurological symptoms: High levels of phenylalanine can cause brain damage, leading to intellectual disability, seizures, and other neurological problems [2]. By reducing phenylalanine levels, sapropterin can help to prevent or alleviate these symptoms [1].
2. Behavioral symptoms: PKU can cause behavioral problems, such as hyperactivity, aggression, and depression [2]. By reducing phenylalanine levels, sapropterin may help to improve these symptoms [1].
3. Skin symptoms: PKU can cause a musty or mouse-like odor of the skin, urine, and breath [2]. By reducing phenylalanine levels, sapropterin may help to alleviate these symptoms [1].

It is important to note that sapropterin is not effective for all patients with PKU, and its effectiveness can vary depending on the type and severity of the PAH deficiency [1]. Therefore, it is essential to consult with a healthcare provider to determine whether sapropterin is appropriate for a particular patient.

Sources:

1. DrugPatentWatch.com. (n.d.). Sapropterin. Retrieved from <https://www.drugpatentwatch.com/drugs/sapropterin>
2. Mayo Clinic. (2021). Phenylketonuria (PKU). Retrieved from <https://www.mayoclinic.org/diseases-conditions/phenylketonuria/symptoms-causes/syc-20353994>