Brand Name
Sephience
Generic Name
Sepiapterin
View Brand Information FDA approval date: July 28, 2025
Form: Powder
What is Sephience (Sepiapterin)?
SEPHIENCE is indicated for the treatment of hyperphenylalaninemia in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria . SEPHIENCE is to be used in conjunction with a phenylalanine -restricted diet. SEPHIENCE is a phenylalanine hydroxylase activator indicated for the treatment of hyperphenylalaninemia in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria . SEPHIENCE is to be used in conjunction with a phenylalanine - restricted diet.
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Related Clinical Trials
A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin
Summary: The main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatment is initiated in early childhood.
A Phase 3 Open-Label Study of PTC923 (Sepiapterin) in Phenylketonuria
Summary: The main purpose of this study is to evaluate the long-term safety of PTC923 in participants with phenylketonuria, and to evaluate the changes from baseline in dietary phenylalanine (Phe)/protein consumption.
Related Latest Advances
Brand Information
Sephience (sepiapterin)
1INDICATIONS AND USAGE
SEPHIENCE is indicated for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive phenylketonuria (PKU). SEPHIENCE is to be used in conjunction with a phenylalanine (Phe)-restricted diet.
2DOSAGE FORMS AND STRENGTHS
Oral powder: 250 mg or 1,000 mg sepiapterin as yellow to orange powder in a unit-dose packet.
3CONTRAINDICATIONS
None.
4ADVERSE REACTIONS
The following clinically significant adverse reactions are described elsewhere in the labeling:
- Increased Bleeding
- Hypophenylalaninemia
4.1Clinical Trials Experience
Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice.
The safety of SEPHIENCE was evaluated in Trial 1 [Part 1 (open label); Part 2, (placebo-controlled)]; and Trial 2 (open-label). The two trials included a total of 215 SEPHIENCE-treated patients with PKU: 10 (5%) were <2 years old, 118 (55%) were ≥2 and <17 years old, and 87 (40%) were ≥17 years old. All patients received SEPHIENCE from 7.5 mg/kg/day up to 60 mg/kg/day and the median duration of treatment (in weeks) was 25.5
Trial 1
Trial 1 included a total of 157 patients (85 male and 72 female, aged 1 year to 61 years old) with PKU across both parts of the trial. The patients received dosages from 20 mg/kg up to 60 mg/kg daily and the median duration of treatment was 8 weeks.
Table 4 lists the most common adverse reactions that were reported in ≥2% of patients treated with SEPHIENCE and greater than that of the placebo group in Part 2 of Trial 1.
Adverse reactions were similar across both adult and pediatric populations except for hypophenylalaninemia (
Description of Selected Adverse Reactions
Increased Bleeding
In Trial 1 Part 2, a case of heavy menstrual bleeding was reported in a SEPHIENCE-treated patient. Less than 2% of patients in Trial 2 experienced increased bleeding, which included heavy menstrual bleeding, non-traumatic superficial hematomas, and prolonged bleeding.
Hypophenylalaninemia
In Trial 1 Part 2, hypophenylalaninemia was seen in 5% (2/37) of sepiapterin-treated pediatric patients and in no adult patients. Some pediatric patients in Trial 2 had multiple low blood Phe levels.
5DESCRIPTION
SEPHIENCE (sepiapterin) contains the drug substance sepiapterin, a PAH activator. Sepiapterin is a yellow to orange powder. Sepiapterin is slightly soluble in water with the solubility at 1.4 mg/mL.
The chemical name of sepiapterin is
SEPHIENCE oral powder contains either 250 mg or 1,000 mg of sepiapterin to be administered orally. The inactive ingredients are: colloidal silicon dioxide, croscarmellose sodium, isomalt, magnesium stearate, mannitol, microcrystalline cellulose, sucralose, and xanthan gum.
6HOW SUPPLIED/STORAGE AND HANDLING
How Supplied
SEPHIENCE (sepiapterin) oral powder is supplied as a yellow to orange powder in a unit-dose heat-sealed laminated aluminum foil packet. Each packet contains:
250 mg sepiapterin per packet:
1,000 mg sepiapterin per packet:
7PATIENT COUNSELING INFORMATION
Advise the patient and/or caregiver to read the FDA-approved patient labeling (Patient Information and Instructions for Use).
Increased Bleeding
Inform the patient about the increased risk of bleeding associated with SEPHIENCE and to follow up with his/her healthcare provider if he/she experiences any symptoms of increased bleeding.
Drug Interactions
Advise the patient to inform his/her healthcare provider if he/she is taking, or plan to take, any prescription or over-the-counter medications and supplements because of the potential for drug interactions
Feces Discoloration
Advise patients that SEPHIENCE may cause feces to have a yellow or orange discoloration.
Manufactured by
Manufactured for:
8PRINCIPAL DISPLAY PANEL - NDC: 52856-201-03 - 250mg Carton Label
9PRINCIPAL DISPLAY PANEL - NDC: 52856-201-01 - 250mg Sachet Label
10PRINCIPAL DISPLAY PANEL - NDC: 52856-201-01 - 250mg Sachet Foil
11PRINCIPAL DISPLAY PANEL - NDC: 52856-301-03 - 1000mg Carton Label
12PRINCIPAL DISPLAY PANEL - NDC: 52856-301-01 - 1000mg Sachet Label
13PRINCIPAL DISPLAY PANEL - NDC: 52856-301-01 - 1000mg Sachet Foil
