Sephience 250 mg/1
SEPIAPTERIN · POWDER · allphamed Pharbil Arzneimittel GmbH
Sepiapterin is an oral powder medication used to treat sepiapterin reductase deficiency, a rare genetic disorder affecting the production of neurotransmitters in the brain. Each dose contains 250 mg of the active ingredient.
Key Facts
- Brand Name
- Sephience
- Generic Name
- SEPIAPTERIN
- NDC Code (Product)
60468-007- Manufacturer
- allphamed Pharbil Arzneimittel GmbH
- Strength
- 250 mg/1
- Dosage Form
- POWDER
- Route
- ORAL
- Marketing Status
- Application #
- NDA219666
- Marketing Start
- 07/28/2025
Recall History
No Recall HistoryFull Prescribing Information
Indications & Usage
1 INDICATIONS 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. SEPHIENCE is a phenylalanine hydroxylase (PAH) activator 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. ( 1 )
Dosage & Administration
2 DOSAGE AND ADMINISTRATION Patients treated with SEPHIENCE should be on a dietary protein and a Phe-restricted diet. ( 2.1 ) Administer SEPHIENCE orally once daily with food. ( 2.2 ) The recommended starting dosage of SEPHIENCE is: ( 2.2 ) Age SEPHIENCE (mg/kg) per day Less than 6 months 7.5 mg/kg 6 months to less than 1 year 15 mg/kg 1 year to less than 2 years 30 mg/kg 2 years and older 60 mg/kg Important Administration Information: prepare SEPHIENCE calculated daily doses of < 1,000 mg as a liquid mixture (25 mg/mL), and administer exact prescribed dose volume (mL). ( 2.2 , 2.3 ) See full prescribing information for complete preparation and administration instructions. ( 2.3 ) 2.1 Important Recommendation Prior to SEPHIENCE Treatment Treatment with SEPHIENCE should be directed by physicians knowledgeable in the management of PKU. Biochemical response to SEPHIENCE treatment cannot generally be pre-determined by laboratory testing (e.g., molecular testing), and should be determined through a therapeutic evaluation of SEPHIENCE [see Dosage and Administration ( 2.2 ) and Clinical Studies ( 14.1 )]. Obtain baseline blood Phe concentration before initiating treatment. All patients wi…
Contraindications
4 CONTRAINDICATIONS None. None. ( 4 )
Drug Interactions
7 DRUG INTERACTIONS Dihydrofolate Reductase (DHFR) Inhibitors : Avoid concomitant use (e.g., trimethoprim, methotrexate, trimetrexate, pemetrexed, pralatrexate, raltitrexed, or piritrexim). ( 7.1 ) Sepiapterin Reductase (SR) Inhibitors : Avoid concomitant use (e.g. sulfasalazine or sulfamethoxazole). ( 7.1 ) Interaction with Levodopa : Monitor patients for a change in neurologic status. ( 7.2 ) Drugs Affecting Nitric Oxide-Mediated Vasorelaxation : Potential for vasorelaxation; monitor blood pressure (e.g., PDE-5 inhibitors). ( 7.2 ) 7.1 Effects of Other Drugs on SEPHIENCE Avoid concomitant use of drugs known to inhibit folate synthesis dihydrofolate reductase (DHFR) (e.g., trimethoprim, methotrexate, trimetrexate, pemetrexed, pralatrexate, raltitrexed, and piritrexim) while taking SEPHIENCE. Concomitant administration of such drugs may reduce sepiapterin metabolism to tetrahydrobiopterin (BH 4 ). If concomitant use is not avoidable, monitor blood Phe levels. Avoid concomitant use of sepiapterin reductase (SR) inhibitors with SEPHIENCE. Concomitant administration of such drugs may reduce sepiapterin metabolism to BH 4 . If concomitant use is not avoidable, monitor blood Phe levels.…
Adverse Reactions
6 ADVERSE REACTIONS The following clinically significant adverse reactions are described elsewhere in the labeling: Increased Bleeding [see Warnings and Precautions ( 5.1 )] . Hypophenylalaninemia [see Warnings and Precautions ( 5.2 )] . Most common adverse reactions with SEPHIENCE (≥2% and greater than placebo) were diarrhea, headache, abdominal pain, hypophenylalaninemia, feces discoloration, and oropharyngeal pain. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact PTC Therapeutics, Inc. at 1-866-562-4620 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical 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…
Frequently Asked Questions
What is Sephience used for?
Sepiapterin is an oral powder medication used to treat sepiapterin reductase deficiency, a rare genetic disorder affecting the production of neurotransmitters in the brain. Each dose contains 250 mg of the active ingredient.
Is Sephience a controlled substance?
Sephience is not classified as a controlled substance by the DEA.
What is the generic name for Sephience?
The generic name for Sephience is SEPIAPTERIN. There are no other listed brand versions of SEPIAPTERIN.
What is the NDC code for Sephience 250 mg/1?
The NDC (National Drug Code) for Sephience 250 mg/1 is 60468-007, listed by allphamed Pharbil Arzneimittel GmbH.
Other Sephience Dosages
Not medical advice. Always consult your doctor or pharmacist before making any medication decisions.
Data from openFDA · Public domain (CC0 1.0)