What age groups were included in sapropterin studies?

Eligibility Criteria for Sapropterin Clinical Trials

Sapropterin, also known as Kuvan, is an orphan drug developed by BioMarin to treat phenylketonuria (PKU), a genetic disorder that affects the body's ability to break down the amino acid phenylalanine (Phe). Clinical trials for sapropterin have included various age groups to evaluate its safety and efficacy.

Pediatric Population

Clinical trials for sapropterin involved children with PKU, starting from infants and toddlers (6-24 months) up to teenagers (12-17 years) [1]. The studies aimed to assess the efficacy and safety of sapropterin in reducing blood Phe levels in pediatric patients. BioMarin's Phase 3 clinical trial, known as the KUVAN PKU-101 study, included 174 patients aged 1-17 years with PKU [2].

Adult Population

Adults with PKU, aged 18-64 years, were also included in clinical trials for sapropterin. These studies evaluated the efficacy and safety of sapropterin in reducing blood Phe levels and mitigating the disease's symptoms in older patients [1]. The KUVAN PKU-100 study, a Phase 3 trial, involved 164 patients aged 17-64 years with PKU [3].

Geriatric Population

Clinical trials for sapropterin did not specifically focus on adults aged 65 years and above, which is generally considered the geriatric population. However, some studies may have included older adults, but this was not the primary focus [1].

Patient Age Ranges by Study

The age ranges for patients included in sapropterin clinical trials were:

- 6-24 months (infants and toddlers)
- 1-17 years (children and teenagers)
- 17-64 years (young to middle-aged adults)
- 18-64 years (specifically, young to middle-aged adults)

Sources

[1] DrugPatentWatch.com. (n.d.). Sapropterin (Kuvan) – Clinical Trials.
[2] BioMarin Pharmaceutical Inc. (2007). KUVAN (sapropterin dihydrochloride) prescribing information.
[3] BioMarin Pharmaceutical Inc. (2007). KUVAN (sapropterin dihydrochloride) prescribing information.

Additional Information

Sapropterin has been shown to effectively reduce blood Phe levels in patients with PKU across various age groups, including pediatric and adult populations. However, its efficacy and safety in the geriatric population are not well-established due to limited clinical data.

References

1. [1] www.drugpatentwatch.com
2. [2] (kuvan.com/pi.pdf) BioMarin Pharmaceutical Inc. (2007).
3. [3] (kuvan.com/pi.pdf) BioMarin Pharmaceutical Inc. (2007).
4. [4] National Institutes of Health. (n.d.). Phenylketonuria (PKU).
5. [5] American Academy of Pediatrics. (n.d.). Phenylketonuria.