About sapropterin di hydrochloride
Synthetic tetrahydrobiopterin (BH4)
Used for
- Helps lower blood phenylalanine in responsive phenylketonuria (PKU).
- Treats high phenylalanine in certain BH4-responsive disorders.
Usual adult dose
Usually 10–20 mg/kg once daily for PKU; dosing is individualized and may differ for BH4 disorders.
Usual dose in children
Usually 10–20 mg/kg once daily for PKU; specialist-directed dosing for BH4 disorders.
Common side effects
- Headache
- Runny or stuffy nose
- Diarrhea or stomach pain
- Nausea or vomiting
Important warnings
- Only works for some people; response should be checked with blood phenylalanine tests.
- Continue prescribed dietary phenylalanine restriction unless the clinician changes it.
- Low phenylalanine can occur; monitor levels and adjust diet or dose.
- Seek urgent care for signs of severe allergic reaction.
Pregnancy
Human pregnancy data are limited; use only when the expected benefit justifies potential risks.
Breastfeeding
It is unknown whether sapropterin passes into human milk; weigh benefits and potential risks.
Interactions
- Levodopa may increase the risk of nervous-system effects, including seizures.
- Medicines affecting folate metabolism, such as methotrexate, may alter response.
- Phosphodiesterase-5 inhibitors may increase blood-pressure-lowering effects.
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 It may be incomplete or out of date.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.