Sephience
What Sephience is used for
Treatment of hyperphenylalaninaemia (HPA) in adult and paediatric patients with phenylketonuria (PKU).
Approved use, as published by the European Medicines Agency.
What the labels mean
- Orphan medicine: for a rare disease (affecting no more than 5 in 10,000 people in the EU).
Key facts
- Brand
- Sephience
- Active substance
- sepiapterin
- Disease area
- Phenylketonurias
- Medicine group
- Other alimentary tract and metabolism products
- ATC code
- A16AX28
- Marketing authorisation holder
- PTC Therapeutics International Limited
- Authorised in the EU since
- 2025
Official information
In the USA
Same active substance: Sephience
Specialist medicine: started and supervised by a specialist doctor or hospital. This page is a plain-language summary of public official information, not medical advice; follow your doctor and the official product information.
Common questions
What is Sephience used for?
Treatment of hyperphenylalaninaemia (HPA) in adult and paediatric patients with phenylketonuria (PKU).
What is the active substance in Sephience?
Sephience contains sepiapterin.
Who makes Sephience?
The EU marketing authorisation holder is PTC Therapeutics International Limited.
Is Sephience an orphan medicine?
Yes. Sephience is designated an orphan medicine in the EU, for a rare disease (Phenylketonurias).
Source: European Medicines Agency medicines data (ema.europa.eu), reproduced with acknowledgement of the source. Page made 5 November 2026 by Satya Babu.
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