About sapropterin di hydrochloride
Tetrahydrobiopterin (BH4) replacement therapy
Used for
- Helps lower blood phenylalanine in some people with phenylketonuria (PKU).
- Treats hyperphenylalaninemia caused by tetrahydrobiopterin deficiency.
Usual adult dose
PKU: usually 10–20 mg/kg once daily. BH4 deficiency: usually 2–5 mg/kg daily, adjusted by response.
Usual dose in children
Same weight-based doses as adults; used in children, including infants, under specialist monitoring.
Common side effects
- Headache
- Runny or stuffy nose
- Diarrhea
- Abdominal pain or nausea
Important warnings
- Requires regular blood phenylalanine monitoring and dietary management.
- Not everyone with PKU responds; assess response during treatment.
- Low phenylalanine can occur, especially in young children or with restricted diets.
- Use cautiously with medicines affecting folate pathways or nitric oxide signaling.
Pregnancy
Limited information; maintaining controlled phenylalanine levels is important during pregnancy. Specialist monitoring is needed.
Breastfeeding
Limited information; discuss use with a healthcare professional, balancing treatment needs and infant exposure.
Interactions
- Levodopa may increase movement-related side effects, including seizures or irritability.
- Medicines inhibiting dihydrofolate reductase may affect response.
- Phosphodiesterase-5 inhibitors may increase effects on nitric oxide signaling.
AI-generated from general medical knowledge, not from this product's official leaflet. It may be incomplete or out of date. Not medical advice — ask a pharmacist or doctor before making any decision.