Source: European Medicines Agency (EU) Revision Year: 2025 Publisher: PTC Therapeutics International Limited, Unit 1, 52-55 Sir John Rogerson's Quay, Dublin 2, D02 NA07, Ireland
Sephience 250 mg oral powder in sachet.
Sephience 1 000 mg oral powder in sachet.
| Pharmaceutical Form |
|---|
|
Oral powder. Yellow to orange powder. |
Sephience 250 mg oral powder in sachet:
Each sachet contains 250 mg of sepiapterin.
Sephience 1 000 mg oral powder in sachet:
Each sachet contains 1 000 mg of sepiapterin.
Excipient(s) with known effect:
Sephience 250 mg oral powder in sachet: Each sachet contains 400 mg of isomalt.
Sephience 1 000 mg oral powder in sachet: Each sachet contains 1 600 mg of isomalt.
For the full list of excipients, see section 6.1.
| Active Ingredient |
|---|
|
Sepiapterin is a natural precursor of the enzymatic co-factor BH4, a critical co-factor for phenylalanine hydroxylase (PAH). Sepiapterin acts as a dual pharmacological chaperone (sepiapterin and BH4 each with its own binding affinity to variant PAH), including PAH variants commonly found in PKU and known to be insensitive to BH4, to improve the activity of the defective PAH enzyme, achieving a high concentration of BH4 intracellularly. |
| List of Excipients |
|---|
|
Microcrystalline cellulose (E460) |
Heat-sealed laminated aluminium foil sachet: Polyethylene terephthalate, white extruded polyethylene (polyester/foil bond), aluminium foil (moisture barrier), and heat-sealed ionomeric resin (adhesive).
Each carton contains 30 unit-dose sachets.
PTC Therapeutics International Limited, Unit 1, 52-55 Sir John Rogerson's Quay, Dublin 2, D02 NA07, Ireland
EU/1/25/1939/001
EU/1/25/1939/002
| Drug | Countries | |
|---|---|---|
| SEPHIENCE | Austria, Lithuania, United States |
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