Sephience

Opinion

EMA has issued an opinion on this medicine

sepiapterin

Overview

On 25 April 2025, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Sephience ¹, intended for the treatment of hyperphenylalaninaemia in adults and children with phenylketonuria (PKU).

The applicant for this medicinal product is PTC Therapeutics International Limited.

Sephience will be available as a 250 mg and 1000 mg oral powder. The active substance of Sephience is sepiapterin, an alimentary tract and metabolism product (ATC code: A16AX28). It is a natural precursor of tetrahydrobiopterin, a critical co‑factor of phenylalanine hydroxylase. Sepiapterin has a dual pharmacological activity, acting as a chaperone in case of a misfolded phenylalanine hydroxylase improving the enzyme’s conformational stability as well as by increasing the intracellular concentrations of tetrahydrobiopterin, thereby reducing blood phenylalanine levels.

The benefits of Sephience are its ability to significantly reduce blood phenylalanine levels after 6 weeks versus placebo, as observed in a double-blind, randomised, placebo-controlled clinical study which included patients of all ages with PKU.

The most common side effects are upper respiratory tract infection, headache, diarrhoea, abdominal pain, hypophenylalaninaemia and discoloration of faeces.

The full indication is:

Sephience is indicated for the treatment of hyperphenylalaninaemia (HPA) in adult and paediatric patients with phenylketonuria (PKU).

Sephience must be initiated and supervised by a physician experienced in the treatment of PKU.

Detailed recommendations for the use of this product will be described in the summary of product characteristics (SmPC), which will be published on the EMA website in all official European Union languages after the marketing authorisation has been granted by the European Commission.

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¹ This product was designated as an orphan medicine during its development. EMA will now review the information available to date to determine if the orphan designation can be maintained.

CHMP summary of positive opinion for Sephience

AdoptedReference Number: EMA/CHMP/132388/2025

Product details

• Name of medicine:
  Sephience
• Active substance:
  sepiapterin
• International non-proprietary name (INN) or common name:
  sepiapterin
• Therapeutic area (MeSH):
  Phenylketonurias
• Anatomical therapeutic chemical (ATC) code:
  A16A
• EMA product number:
  EMEA/H/C/006331
• Orphan:

  This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

• Marketing authorisation applicant:
  PTC Therapeutics International Limited
• Opinion adopted:
  25/04/2025
• Opinion status:
  Positive