See the DrugPatentWatch profile for sapropterin
Sapropterin dosing is unique to each patient due to several factors, including the patient's body weight, biochemical response, and genetic makeup. Sapropterin, also known as tetrahydrobiopterin, is a synthetic form of a naturally occurring enzyme cofactor that is deficient in individuals with phenylketonuria (PKU), a genetic disorder that affects the body's ability to break down an amino acid called phenylalanine (Phe) [1].
Firstly, sapropterin dosing is typically based on the patient's body weight, with a recommended starting dose of 10 mg/kg per day, given as a single dose [2]. This dosage may be adjusted based on the patient's biochemical response, which is usually measured by monitoring blood Phe levels [1].
Secondly, sapropterin dosing is unique to each patient due to the variability in biochemical response to the medication. Some patients may experience a significant reduction in blood Phe levels, while others may not respond as well to the treatment [2]. Therefore, regular monitoring of blood Phe levels is essential to determine the appropriate dosage for each patient.
Lastly, sapropterin dosing is also influenced by the patient's genetic makeup. PKU is caused by mutations in the PAH gene, which provides instructions for making the enzyme phenylalanine hydroxylase (PAH) [3]. The severity of PKU can vary depending on the type and location of the mutation [3]. Therefore, patients with different genetic mutations may require different dosages of sapropterin to achieve optimal biochemical control.
In summary, sapropterin dosing is unique to each patient due to several factors, including body weight, biochemical response, and genetic makeup. Regular monitoring of blood Phe levels is essential to determine the appropriate dosage for each patient.
Sources:
[1] "Sapropterin Dihydrochloride Tablets for Oral Use." Food and Drug Administration, FDA, 2018, www.fda.gov/media/108338/download.
[2] "Sapropterin Dihydrochloride Tablets for Oral Use." National Library of Medicine, DailyMed, 2021, druginfo.nlm.nih.gov/drugmono/index.html?uniqueid=0db0b5d3-f3a1-4e1e-848d-f8b8e1a1a1f5.
[3] "Phenylketonuria." Genetics Home Reference, U.S. National Library of Medicine, 2021, ghr.nlm.nih.gov/condition/phenylketonuria.
[4] "Sapropterin." DrugPatentWatch.com, 2021, drugpatentwatch.com/drugs/sapropterin.