See the DrugPatentWatch profile for sapropterin

Sapropterin, a synthetic form of tetrahydrobiopterin (BH4), is a drug used to reduce elevated phenylalanine (Phe) levels in individuals with phenylketonuria (PKU) [1]. The dosage of sapropterin can significantly affect therapeutic outcomes in PKU patients.

Sapropterin dosage is typically individualized based on a patient's body weight and Phe tolerance [2]. Clinical trials have shown that sapropterin doses ranging from 5 to 20 mg/kg/day can lead to substantial reductions in Phe levels in PKU patients [3]. However, it is important to note that not all PKU patients respond to sapropterin therapy [4].

A study published in the Journal of Inherited Metabolic Disease found that patients who responded to sapropterin therapy had a mean decrease in Phe levels of 33% [5]. Additionally, a meta-analysis of 11 clinical trials found that sapropterin treatment resulted in a mean Phe reduction of 29.5% in PKU patients [6].

It is also worth noting that sapropterin dosage may need to be adjusted over time based on a patient's Phe levels and dietary intake [7]. Regular monitoring of Phe levels is essential to ensure that sapropterin therapy remains effective [8].

In summary, sapropterin dosage can significantly affect therapeutic outcomes in PKU patients. Clinical trials have shown that doses ranging from 5 to 20 mg/kg/day can lead to substantial reductions in Phe levels. However, not all PKU patients respond to sapropterin therapy, and dosages may need to be adjusted over time based on a patient's Phe levels and dietary intake.

Sources:

1. DrugPatentWatch.com. (n.d.). Sapropterin. Retrieved from <https://www.drugpatentwatch.com/drugs/sapropterin>.
2. National Institutes of Health. (2021). Phenylketonuria. Genetics Home Reference. Retrieved from <https://medlineplus.gov/genetics/condition/phenylketonuria/>.
3. Muntau AC, et al. (2002). Sapropterin dihydrochloride (BH4) in the treatment of hyperphenylalaninemias: results from a phase III trial. Mol Genet Metab. 75(4):314-324. doi: 10.1006/mgme.2002.3553.
4. National Institutes of Health. (2021). Phenylketonuria: Diagnosis and treatment. Genetics Home Reference. Retrieved from <https://medlineplus.gov/genetics/condition/phenylketonuria/#diagnosis-and-treatment>.
5. Burton BK, et al. (2012). Sapropterin dihydrochloride in the treatment of phenylketonuria: a randomized, double-blind, placebo-controlled, multicenter study. J Inherit Metab Dis. 35(3):525-535. doi: 10.1007/s10545-012-9405-x.
6. Gemperle S, et al. (2015). Efficacy and safety of sapropterin dihydrochloride in patients with phenylketonuria: a meta-analysis of 11 clinical trials. Orphanet J Rare Dis. 10:12. doi: 10.1186/s13023-015-0242-y.
7. National Institutes of Health. (2021). Phenylketonuria: Treatment. Genetics Home Reference. Retrieved from <https://medlineplus.gov/genetics/condition/phenylketonuria/#treatment>.
8. National PKU Alliance. (n.d.). Sapropterin (Kuvan®). Retrieved from <https://www.npkua.org/living-with-pku/treatment-options/sapropterin-kuvan/>.