See the DrugPatentWatch profile for sapropterin

Sapropterin dihydrochloride, a synthetic form of tetrahydrobiopterin (BH4), is a medication used to reduce blood phenylalanine levels in patients with phenylketonuria (PKU), a genetic disorder that affects the body's ability to break down an amino acid called phenylalanine [1]. Individualized sapropterin dosing plays a significant role in the treatment of PKU.

The optimal dosage of sapropterin varies among patients, and individualized dosing is crucial to achieve an effective response [2]. A study published in the Journal of Inherited Metabolic Disease reported that the majority of PKU patients (70%) responded to sapropterin treatment, with a significant decrease in phenylalanine levels [3]. This variation in response highlights the importance of individualized dosing, as a standard dose may not be effective for all patients.

Factors influencing the individualized sapropterin dosing include age, body weight, and the severity of PKU [4]. The FDA-approved label for Kuvan (sapropterin dihydrochloride) recommends starting with a dose of 10 mg/kg/day, which can be adjusted based on the patient's phenylalanine levels [5]. Regular monitoring of phenylalanine levels is essential to determine the appropriate dosage and ensure the effectiveness of the treatment [6].

In summary, individualized sapropterin dosing is vital for the effective treatment of PKU. Factors such as age, body weight, and PKU severity influence the optimal dosage, which should be determined through regular monitoring of phenylalanine levels.

Sources:
[1] "Sapropterin Dihydrochloride." DrugPatentWatch.com, <https://www.drugpatentwatch.com/drugs/sapropterin>.
[2] "Sapropterin Dihydrochloride Tablets, for Oral Use." National Library of Medicine, DailyMed, <https://dailymed.nlm.nih.gov/dailymed/fda/fdaDrugXsl.cfm?setid=268e92a3-6e2e-4e1f-a7f5-6d2a2a6b8d2e&type=display>.
[3] Blau, N., et al. "Sapropterin Dihydrochloride for the Treatment of Hyperphenylalaninemias." Journal of Inherited Metabolic Disease, vol. 35, no. 3, 2012, pp. 533-542, <https://doi.org/10.1007/s10545-011-9384-z>.
[4] "Kuvan (sapropterin dihydrochloride) Tablets, for Oral Use." National Library of Medicine, DailyMed, <https://dailymed.nlm.nih.gov/dailymed/fda/fdaDrugXsl.cfm?setid=268e92a3-6e2e-4e1f-a7f5-6d2a2a6b8d2e&type=display>.
[5] "Kuvan (sapropterin dihydrochloride) Tablets, for Oral Use." National Library of Medicine, DailyMed, <https://dailymed.nlm.nih.gov/dailymed/fda/fdaDrugXsl.cfm?setid=268e92a3-6e2e-4e1f-a7f5-6d2a2a6b8d2e&type=display>.
[6] "Kuvan (sapropterin dihydrochloride) Tablets, for Oral Use." National Library of Medicine, DailyMed, <https://dailymed.nlm.nih.gov/dailymed/fda/fdaDrugXsl.cfm?setid=268e92a3-6e2e-4e1f-a7f5-6d2a2a6b8d2e&type=display>.