Active Ingredient: Sepiapterin
Sepiapterin is indicated for the treatment of hyperphenylalaninaemia (HPA) in adult and paediatric patients with phenylketonuria (PKU).
For this indication, competent medicine agencies globally authorize below treatments:
For:
Regimen A, in case that patient age in months is < 6
Oral, 7.5 milligrams sepiapterin per kilogram of body weight, once daily.
Regimen B, in case that patient age in months is ≥ 6 and patient age in months is < 12
Oral, 15 milligrams sepiapterin per kilogram of body weight, once daily.
Regimen C, in case that patient age in months is ≥ 12 and patient age in years is < 2
Oral, 30 milligrams sepiapterin per kilogram of body weight, once daily.
Regimen D, in case that patient age in years is ≥ 2
Oral, 60 milligrams sepiapterin per kilogram of body weight, once daily.
The recommended dose (mg/kg/day) of sepiapterin to be administered orally once daily is based on age and body weight (see table). The maximum recommended dose is 60 mg/kg/day. The recommended dose of sepiapterin in patients ≥2 years of age is 60 mg/kg/day. However, the dose may be adjusted to a lower dose if the treating physician considers it necessary or appropriate.
Recommended dose based on patient's age and body weight:
| Age | Recommended dose (mg/kg) of sepiapterin per day |
|---|---|
| 0 to <6 months | 7.5 mg/kg/day |
| 6 to <12 months | 15 mg/kg/day |
| 12 months to <2 years | 30 mg/kg/day |
| ≥2 years | 60 mg/kg/day |
A missed dose should be taken as soon as possible. The normal dosing schedule should be resumed the following day.
In the pivotal Phase 3 clinical study, a threshold of 15% or greater reduction in blood phenylalanine (Phe) levels was utilised for determination of response.
No controlled efficacy and safety data are available in patients who do not experience a reduction of 15% or greater reduction in blood Phe levels after receiving sepiapterin for 14 days.
The determination of responsiveness for a patient with PKU and the discontinuation of the medicinal product is at the discretion of the treating physician.
The safety and efficacy of sepiapterin in patients 65 years of age and older have not been established. Caution should be exercised when prescribing in patients 65 years of age and older.
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