ARTG Entry
SEPHIENCE
ARTG entry for SEPHIENCE (sepiapterin), ARTG 453665 — Product Information, dosage form, registration history. Compiled by arcimedes.
- Sponsor: Ptc Therapeutics
- Active ingredient: sepiapterin
- Therapeutic area: Rare Disease
What it is
SEPHIENCE is an oral powder containing sepiapterin, available in 250 mg and 1000 mg sachets. The product is a yellow to orange powder for oral use. SEPHIENCE is subject to additional monitoring in Australia to allow quick identification of new safety information.
Approved indications
— Treatment of hyperphenylalaninae mia (HPA) in adult and paediatric patients with phenylketonuria (PKU).
Dosing overview
The recommended dose of SEPHIENCE, administered orally once daily, is based on age and body weight. The maximum recommended dose in paediatric patients aged 2 years and older and adults is 60 mg/kg/day. For patients 0 to less than 6 months, the dose is 7.5 mg/kg/day; 6 to less than 12 months, 15 mg/kg/day; 12 months to less than 2 years, 30 mg/kg/day; and 2 years and older, 60 mg/kg/day. SEPHIENCE should be administered orally once a day with food using mg/kg/day dosing.
Key safety warnings
Patients treated with SEPHIENCE should undergo regular clinical assessments to align with their healthcare provider on appropriate dietary phenylalanine intake, such as monitoring of blood phenylalanine and tyrosine levels and nutritional intake. Co-administering SEPHIENCE with dihydrofolate reductase (DHFR) inhibitors such as trimethoprim, methotrexate, pemetrexed and pralatrexate may require more frequent monitoring of blood phenylalanine levels because these drugs may inhibit the enzymatic conversion of sepiapterin to tetrahydrobiopterin (BH4). Caution should be exercised when prescribing SEPHIENCE to patients receiving treatment with levodopa to monitor neurological disorders such as exacerbation of convulsion, increased excitability and irritability, seizures, and exacerbation of seizures. Long-term safety data in patients with PKU are limited.
Contraindications
SEPHIENCE is contraindicated in patients with hypersensitivity to sepiapterin or to any of the excipients.
PBS listing
Information regarding PBS listing is not available in the provided documents.
Regulatory history
SEPHIENCE received its first approval on 17 July 2025. ARTG registration records show SEPHIENCE sepiapterin 1000 mg and 250 mg oral powder sachets were first listed on 2025-07-17. The TGA approved SEPHIENCE for the treatment of hyperphenylalaninaemia (HPA) in adult and paediatric patients with phenylketonuria (PKU) on 14 July 2025, following a positive assessment of its quality, nonclinical, and clinical data.