Sapropterin
Kuvan · ATC code A16AX07
Also known as: Sapropterina, Saproptérine · Brand names: Kuvan, Sapropterin Amarox, Diterin, Fenterin, Kuvamex, Pekutin, Saptera
- Key facts
- Active substance
- Sapropterin
- ATC code
- A16AX07
- Authorisation
- centrally authorised across the EU (EMA)
- Dosage forms
- Kuvan 100 mg soluble tablets · Kuvan 100 mg powder for oral solution / Kuvan 500 mg powder for oral solution
- Marketing authorisation holder
- BioMarin International Limited
- Information as of
- 2026-10-27
Key facts
Sapropterin is a synthetic form of tetrahydrobiopterin (BH4). It treats hyperphenylalaninaemia due to phenylketonuria or BH4 deficiency in responsive patients of all ages.
What it is used for
- Hyperphenylalaninaemia in phenylketonuria (PKU) shown to be responsive, adults and children of all ages
- Hyperphenylalaninaemia in BH4 deficiency shown to be responsive, adults and children of all ages
Dosing
- PKU: start 10 mg/kg body weight once daily; adjust usually between 5 and 20 mg/kg/day.
- BH4 deficiency: start 2 to 5 mg/kg total daily dose; adjust up to 20 mg/kg per day.
- Round the calculated daily dose to the nearest multiple of 100 mg.
- Test phenylalanine and tyrosine one to two weeks after each dose adjustment and monitor frequently.
- Continue phenylalanine-restricted diet; discontinue only under physician supervision.
Contraindications
- Hypersensitivity to the active substance or any excipient
Important warnings
- Continue restricted phenylalanine diet and regular assessment (phenylalanine, tyrosine, nutrient intake, development)
- Low phenylalanine and tyrosine levels in infancy are associated with impaired neurodevelopment
- Phenylalanine levels may rise during illness
- Caution with levodopa (convulsions, increased excitability observed)
- Rebound of phenylalanine levels may occur after stopping
Side effects
- Very common: headache, rhinorrhoea
- Common: hypophenylalaninaemia
- Children below 4 years: most commonly amino acid level decreased, vomiting, rhinitis
- Not known: hypersensitivity reactions and rash
Interactions
- Dihydrofolate reductase inhibitors (e.g. methotrexate, trimethoprim): caution
- Vasodilators acting on NO (e.g. glyceryl trinitrate, isosorbide dinitrate, nitroprusside, molsidomin, PDE-5 inhibitors, minoxidil): caution
- Levodopa: caution due to convulsions
Pregnancy and breastfeeding
- Limited data in pregnancy; too limited to conclude on malformative or foeto-neonatal toxicity.
Summary of the official product information, written with AI on 2026-10-28. Every number was checked automatically against the original text. Not reviewed by a physician – the full official text is authoritative.
Common questions – ask Medelix
For patients
Package leaflet, verbatim
Authorisation and brand names by country
Medicines with exactly this active-substance composition, as listed in the national registers – alphabetical and without ranking. Whether a product is currently available is not shown here.
EU – centrally authorised (all member states)
- KuvanBioMarin International Limited · EMEA/H/C/000943
- Sapropterin Dipharma · genericDipharma Arzneimittel GmbH · EMEA/H/C/005646
Source: European Medicines Agency (EMA), product information
Germany
- Sapropterin AmaroxAmarox Pharma Gmbh · 1 register entry
Source: EMA – Article 57 product data (EU/EEA), as of 2026-10-28
Turkey
- DiterinTRPHARM İLAÇ SAN. TİC. A.Ş. · 1 register entry
- FenterinPOLİFARMA İLAÇ SAN. VE TİC. A.Ş. · 2 register entries
- KuvamexİLKO İLAÇ SAN.VE TİC. A.Ş. · 1 register entry
- KuvanBİOMARİN İLAÇ VE TİCARET LİMİTED ŞİRKETİ · 3 register entries
- PekutinBİLİM İLAÇ SAN. VE TİC. A.Ş. · 1 register entry
- SapteraNOBEL İLAÇ SAN. VE TİC. A.Ş. · 1 register entry
Source: TİTCK – Ruhsatlı Beşeri Tıbbi Ürünler Listesi, as of 2026-10-16
Medelix receives no money from pharmaceutical companies. Brand names are listed alphabetically and without ranking.
Other substances in the same group
Another question about this substance?
Medelix answers clinical questions from guidelines, studies and product information – every claim with its source.
Sources
- European Medicines Agency. Kuvan – EPAR, Product Information. 2026-10-27. https://www.ema.europa.eu/en/documents/product-information/kuvan-epar-product-information_en.pdf
- EMA – Article 57 product data (EU/EEA), 2026-10-28. https://www.ema.europa.eu/en/human-regulatory-overview/post-authorisation/data-medicines-iso-idmp-standards-post-authorisation/public-data-article-57-database
- TİTCK – Ruhsatlı Beşeri Tıbbi Ürünler Listesi, 2026-10-16. https://www.titck.gov.tr/dinamikmodul/85
Editorial: Medelix editorial team · last updated 2026-10-27 · How these pages are made · Report an error
Source: European Medicines Agency (EMA), product information. Not advertising, not a treatment recommendation. For questions about your own treatment, ask your doctor or pharmacist.