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Sapropterin

Kuvan · ATC code A16AX07

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 (Claude, Anthropic) on 2026-10-28. Every number was checked automatically against the original text. Not reviewed by a physician – the full official text is authoritative.

Full official text →

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For patients

Package leaflet, verbatim

Package leaflet for patients →

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.

Another question about this substance?

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Sources

  1. European Medicines Agency. Kuvan – EPAR, Product Information (OF THE TEXT Date of revision of the text: MM/YYYY Detailed i). 2026-10-27. https://www.ema.europa.eu/en/documents/product-information/kuvan-epar-product-information_en.pdf
  2. 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
  3. 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.