Sephience SEPIAPTERIN 1000 mg Powder, 30 packets — NDC 60468-006-03 (Billing 60468-0006-03)
This is a package of 30 packets of Sephience SEPIAPTERIN 1000 mg Powder from allphamed Pharbil Arzneimittel GmbH, marketed since Jul 2025 and currently FDA-listed. It is this product's only package size.
Identity & classification
Regulatory identifiers FDA, NLM and CMS codes for this package
- RxCUI (RxNorm): 2723061
Where does this data come from?
- FDA openFDA NDC Directory · synced Oct 1, 2026
- FDA label on DailyMed · label index refreshed Oct 1, 2026
- RxNorm (NLM RxNav) · catalog refreshed Oct 1, 2026
- Medi-Span GPI (licensed)
- First Databank (licensed) · refreshed Oct 1, 2026
RxNorm drug class
This medicine belongs to the Various alimentary tract and metabolism products class.
Where does this data come from?
- RxClass (NLM) · catalog refreshed Oct 1, 2026
Clinical
Sepiapterin is used to treat hyperphenylalaninemia (HPA; elevated levels of the amino acid phenylalanine) due to phenylketonuria (PKU; disorder where body cannot break down phenylalanine). Sepiapterin is in a class of medications called phenylalanine hydroxylase (PAH) activators. It works by helping the phenylalanine hydroxylase enzyme to break down phenylalanine to tyrosine.
Read the full MedlinePlus article ↗Patient education
Supplement & herbal interactions
Where does this data come from?
- MedlinePlus (NLM) · refreshed Oct 1, 2026
- FDA label on DailyMed · label index refreshed Oct 1, 2026
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Pricing
A drug doesn't have one price. Each row is a different public payment system, and none is what you'd pay at the counter — that depends on your insurance. The ⓘ on each row explains what it measures.
| Price system | Per g | Per package |
|---|---|---|
| Retail pharmacies payNADAC · weekly | Not in the retail survey — common for institutional, discontinued, or low-volume packs. | |
| Medicaid paysCMS SDUD · 12 mo | No recent Medicaid claims on file for this NDC — rare and low-volume NDCs are suppressed in the public data. | |
| Medicare drug plans payPart D · Q2 2026 | $130.26 | — |
Where does this data come from?
- CMS NADAC weekly file
- CMS ASP pricing files · refreshed Sep 20, 2026
- CMS Medicaid State Drug Utilization Data · refreshed Oct 3, 2026
- CMS Part D plan pricing files · refreshed Sep 24, 2026
- VA National Acquisition Center price file
Packaging — all sizes for this product
| Package NDC | Description | Marketing start | Marketing end | Status |
|---|---|---|---|---|
| 60468-0006-03 You're viewing this Main listing | 30 PACKET in 1 CARTON / 1 POWDER in 1 PACKET | 2025-07-28 | — | Active |
Therapeutic equivalents
| Product | Labeler | Pack | NADAC/unit | TE | Status | Price vs. this |
|---|---|---|---|---|---|---|
| Sephience 1000 mg 52856-0301-03 | PTC | 30 packets | — | — | FDA listed | — |
| Sephience 1000 mgthis 60468-0006-03 | allphamed | 30 packets | — | — | FDA listed | — |
Where does this data come from?
- FDA openFDA NDC Directory · synced Oct 1, 2026
- FDA Orange Book · refreshed Sep 3, 2026
- CMS NADAC weekly file
Availability & generic status
We did not find an FDA-approved generic match for this exact strength, form and route. Patent/protection dates below may affect future generic timing.
Why the date isn’t exact: Generic timing can change because patents may be challenged, settled, licensed, added, removed, or worked around with a narrower label — and FDA approval does not always mean a pharmacy can get the generic today.
🛈 What do these terms mean?
- Patent
- Legal protection listed in the Orange Book that may delay generic approval or launch. Issued by the U.S. Patent & Trademark Office.
- Substance patent
- Covers the active drug molecule itself — the hardest to design around. A generic generally can’t launch until it expires.
- Formulation (product) patent
- Covers a specific formulation or dosage form. A generic can sometimes work around it with a different formulation.
- Method-of-use patent
- A patent covering one specific approved use of the drug — not necessarily the whole molecule. A generic can sometimes launch with a “skinny label” that carves out the protected use and keeps the others.
- Skinny label
- A generic label that omits a still-patented use when the FDA allows it — letting a generic reach the market for the unprotected uses.
- Exclusivity
- FDA-granted marketing protection, separate from patents — e.g. 5-yr new chemical entity, 7-yr orphan drug, or a +6-month pediatric extension.
- Paragraph IV
- A generic applicant’s formal challenge to a listed patent. It can potentially lead to earlier generic entry, but often involves litigation or a settlement.
- RLD / RS
- Reference Listed Drug — the brand product the FDA uses as the reference for generic applications. Reference Standard — the product the FDA expects generics to compare against in bioequivalence testing.
- TE / AB rating
- FDA therapeutic-equivalence rating. An AB rating generally means the FDA considers a generic therapeutically equivalent to — and substitutable for — the brand.
- LOE (loss of exclusivity)
- The latest patent or exclusivity currently listed — the loss-of-exclusivity / latest-listed-protection date shown on this page. Paragraph-IV challenges and settlements can move the real date earlier; FDA approval and a manufacturer’s decision to market can move it later.
Built from the FDA Orange Book. The bars above are scaled to each protection’s expiry; the red LOE marker is the last one to lapse.
| Patent | Type | Use code | Expires |
|---|---|---|---|
| US 12257252 ↗ | Method of use | U-4250 | Mar 6, 2042 |
| US 12257252 ↗ | Method of use | U-4250 | Mar 6, 2042 |
| US 12213982 ↗ | Method of use | U-4251 | Sep 4, 2038 |
| US 12213982 ↗ | Method of use | U-4251 | Sep 4, 2038 |
| US 11752154 ↗ | Method of use | U-4251 | Sep 4, 2038 |
| US 11752154 ↗ | Method of use | U-4251 | Sep 4, 2038 |
| US 11072614 ↗ | Drug substance | U-4251 | Apr 16, 2038 |
| US 11072614 ↗ | Drug substance | U-4251 | Apr 16, 2038 |
| Code | What it grants | Expires |
|---|---|---|
| NCE | New Chemical Entity (5-year) | Jul 28, 2030 |
| ODE-542 | Orphan Drug Exclusivity (7-year) | Jul 28, 2032 |
| NCE | New Chemical Entity (5-year) | Jul 28, 2030 |
| ODE-542 | Orphan Drug Exclusivity (7-year) | Jul 28, 2032 |
Is there a generic version of this drug?
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Where does this data come from?
- FDA Orange Book · refreshed Sep 3, 2026
What it looks like
Where does this data come from?
- FDA label on DailyMed · label index refreshed Oct 1, 2026
Inactive Ingredients / Excipients
Inactive ingredients, also called excipients, are components of the drug product other than the active ingredient. They may include fillers, dyes, coatings, preservatives, flavors, or other formulation ingredients.
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ingredient classCode="IACT" elements from the exact product block matched by this NDC. Label-section narrative from DailyMed / the openFDA label index is shown separately when available.- FDA label on DailyMed · label index refreshed Oct 1, 2026
- FDA openFDA NDC Directory · synced Oct 1, 2026
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Manufacturer & labeler
Where does this data come from?
- FDA openFDA NDC Directory · synced Oct 1, 2026
- Drugs@FDA
Full prescribing information FDA SPL
🎯 Indications and 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 and 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 )
2.1Important 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 with PKU who are treated with SEPHIENCE should be on a dietary protein and Phe-restricted diet that is based on blood Phe levels. Patients should undergo regular dietary assessments, including protein and Phe intake, by their healthcare provider [see Dosage and Administration ( 2.2 )].
2.2Recommended Dosage and Administration The recommended starting dosage of SEPHIENCE is based on the patient’s age and is administered orally once daily (see Table 1 ). Administer SEPHIENCE with food [see Clinical Pharmacology ( 12.3 )] . Table 1: Recommended Starting Dosage of SEPHIENCE * in Pediatric and Adult Patients * For calculated daily doses less than 1,000 mg, the final concentration of prepared SEPHIENCE liquid mixture is 25 mg/mL [see Dosage and Administration ( 2.3 )] . ** 60 mg/kg is the maximum daily dose for all patients.
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 Evaluation Period Dosage Titration in Patients Less than 2 Years of Age After initiating treatment at the starting dosage by age ( Table 1 ), check blood Phe levels to determine response to treatment within 2 weeks. If blood Phe does not decrease, SEPHIENCE dosage may be titrated incrementally based on blood Phe levels to a maximum daily dosage of 60 mg/kg.
Existing dietary protein and Phe intake should not be modified during the evaluation period. Discontinuation for Lack of Biochemical Response Discontinue SEPHIENCE in patients whose blood Phe does not decrease after 2 weeks of treatment at the maximum daily dosage of 60 mg/kg. Dosage Modification and Monitoring Monitor blood Phe levels during treatment, and if needed, modify the daily dosage of SEPHIENCE within the range of 7.5 mg/kg to 60 mg/kg and/or dietary protein and Phe intake to ensure adequate blood Phe level control.
Frequent blood Phe monitoring is recommended in the pediatric population [see Warnings and Precautions ( 5.2 )] . Missed Dose A missed dose should be taken as soon as possible but 2 doses should not be administered on the same day. Resume the normal dosing schedule the following day.
2.3Preparation and Administration Instructions Doses Less Than 1,000 mg ( administration based on 25 mg/mL concentration ) Determine the required number of SEPHIENCE packets and the required volume of water or apple juice to achieve a concentration of 25 mg/mL mixture (see Table 2 ). Table 2: Number of SEPHIENCE Packets and Volume to Prepare a SEPHIENCE Mixture of 25 mg/mL for Doses Less than 1,000 mg Abbreviations: mg, milligrams; mL, milliliters a For calculated daily doses less than 1,000 mg, round the dose up to the nearest 250 mg to determine the number of SEPHIENCE packets and prepare each 250 mg packet with 9 mL o… [Excerpted — this section continues on DailyMed.]
💊 Dosage Forms and Strengths ▾
3 DOSAGE FORMS AND STRENGTHS Oral powder: 250 mg or 1,000 mg sepiapterin as yellow to orange powder in a unit-dose packet. Oral Powder: 250 mg ( 3 ) Oral Powder: 1000 mg ( 3 )
⛔ Contraindications ▾
4 CONTRAINDICATIONS None. None. ( 4 )
⚠️ Warnings and Cautions ▾
5 WARNINGS AND PRECAUTIONS Increased Bleeding : SEPHIENCE may increase the risk of bleeding. Consider treatment interruption in patients with active bleeding. ( 5.1 ) Hypophenylalaninemia : Some pediatric PKU patients experienced hypophenylalaninemia; monitor patients blood Phe levels during treatment. ( 5.2 ) Interaction with Levodopa : Seizures, over-stimulation or irritability may occur; monitor patients for a change in neurologic status. ( 5.3 )
5.1Increased Bleeding SEPHIENCE may increase the risk of bleeding. Bleeding events, including superficial hematomas, prolonged bleeding, and heavy menstrual bleeding have occurred in patients treated with SEPHIENCE [see Adverse Reactions ( 6.1 )] . One patient with non-traumatic superficial hematomas and prolonged bleeding was re-challenged at a lower dose of SEPHIENCE with recurrence of symptoms, which led to treatment discontinuation.
The patient experienced symptoms 15 days after initial exposure and two days after rechallenge. The patient had normal blood counts and coagulation studies at the time of the 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 signs of increased bleeding.
Consider treatment interruption with SEPHIENCE in patients with active bleeding.
5.2Hypophenylalaninemia In clinical trials of SEPHIENCE, some pediatric PKU patients experienced hypophenylalaninemia (low blood Phe), including some patients with multiple low blood Phe levels, during treatment with SEPHIENCE [see Adverse Reactions ( 6.1 )] . Prolonged levels of blood Phe that are too low have been associated with catabolism and endogenous protein breakdown, which has been associated with adverse developmental outcomes. Monitor blood Phe levels during treatment and if needed, modify the dosage of SEPHIENCE and/or dietary protein and Phe intake to ensure adequate blood Phe level control.
Frequent blood Phe monitoring is recommended in the pediatric population [see Dosage and Administration ( 2.2 )] .
5.3Interaction with Levodopa In a 10-year post-marketing safety surveillance program for a non-PKU indication using another drug that is a phenylalanine hydroxylase (PAH) activator, 3 patients with underlying neurological disorders experienced seizures, exacerbation of seizures, over-stimulation, and irritability during co-administration with levodopa. Monitor patients who are receiving levodopa for changes in neurological status during treatment with SEPHIENCE [see Drug Interactions ( 7.2 )] .
🤒 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.
6.1Clinical 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 [see Clinical Studies ( 14.1 )] . 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. Table 4: Adverse Reactions for SEPHIENCE in Adult and Pediatric Patients with PKU that Occurred in ≥2% of Sepiapterin-Treated Patients and Greater than Placebo (Trial 1 Part 2) a Includes Abdominal pain; Abdominal pain upper, Abdominal discomfort. Adverse Reaction SEPHIENCE N=56 N (%) Placebo N=54 N (%) Diarrhea 4 (7) 1 (2) Headache 4 (7) 1 (2) Abdominal pain a 3 (5) 1 (2) Hypophenylalaninemia 2 (4) 0 Feces discoloration 2 (4) 0 Oropharyngeal pain 2 (4) 1 (2) Adverse reactions were similar across both adult and pediatric populations except for hypophenylalaninemia ( see Description of Selected Adverse Reactions ).
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.
🔄 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 )
7.1Effects 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.
7.2Effects of SEPHIENCE on Other Drugs SEPHIENCE may increase the availability of tyrosine, a precursor of levodopa. Neurologic events were reported postmarketing in patients receiving another PAH activator and levodopa concomitantly for a non-PKU indication. Monitor patients for a change in neurologic status when levodopa is administered with SEPHIENCE [see Warnings and Precautions ( 5.3 )] .
7.3Drugs Affecting Nitric Oxide-Mediated Vasorelaxation SEPHIENCE and PDE-5 inhibitors induce vasorelaxation, thus concomitant use of SEPHIENCE with PDE-5 inhibitors may reduce blood pressure even further. Monitor for signs and symptoms of hypotension with concomitant use of SEPHIENCE with drugs that affect nitric oxide-mediated vasorelaxation (e.g., PDE-5 inhibitors such as sildenafil, vardenafil, or tadalafil).
👥 Use in Specific Populations ▾
8 USE IN SPECIFIC POPULATIONS
8.1Pregnancy Risk Summary Available data on the use of SEPHIENCE during pregnancy are insufficient to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. Uncontrolled blood Phe concentrations before and during pregnancy are associated with an increased risk of adverse pregnancy outcomes and fetal adverse effects ( see Clinical Considerations ) . In animal reproduction studies, oral administration of sepiapterin to pregnant rats and rabbits during organogenesis at dose exposures up to 9- and 6- times the human exposure at the maximum recommended human dose (MRHD) of 60 mg/kg, respectively, resulted in no adverse developmental effects ( see Data ).
All pregnancies have a background risk of birth defects, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. The background risk of major birth defects and miscarriage in pregnant women with PKU who maintain blood Phe concentrations greater than 600 micromol/L during pregnancy is greater than the corresponding background risk for pregnant women without PKU.
Clinical Considerations Disease-Associated Maternal and/or Embryofetal Risk Uncontrolled blood Phe concentrations before and during pregnancy are associated with an increased risk of adverse outcomes and fetal adverse effects ( see Data ). To reduce the risk of hyperphenylalaninemia-induced fetal adverse effects, blood Phe concentrations should be maintained between 120 and 360 micromol/L during pregnancy and during the 3 months before conception [see Dosage and Administration ( 2.2 )] . Data Human Data Available data from the Maternal Phenylketonuria Collaborative Study on 468 pregnancies and 331 live births in pregnant women with PKU demonstrated that uncontrolled Phe concentrations above 600 micromol/L are associated with an increased risk for major birth defects (including microcephaly, major cardiac malformations), intrauterine fetal growth retardation, and future intellectual disability with low IQ.
Animal Data In an embryo-fetal development study in rats, SEPHIENCE was administered at dose levels of 100, 300, or 1,000 mg/kg/day via oral gavage to pregnant rats during the period of organogenesis from gestation day (GD) 7 to GD 17. There were no maternal or embryo-fetal developmental toxicities noted at doses up to 1,000 mg/kg/day (9-fold the human AUC 0-24 at the MRHD). In an embryo-fetal development study in pregnant rabbits, SEPHIENCE was administered at dose levels of 100, 300, or 1,000 mg/kg/day via oral gavage to pregnant rabbits during the period of organogenesis from gestation day (GD) 7 to GD 19.
There were no maternal or embryo-fetal developmental toxicities at doses up to 1,000 mg/kg/day (6-fold the human AUC 0-24 at the MRHD). In the pre- and post-natal development study in rats, SEPHIENCE was administered at dose levels of 30, 100, and 300 mg/kg/day via oral gavage once daily to pregnant rats from GD 6 to lactation day 20. SEPHIENCE did not induce effects on maternal reproductive function or on developmental and reproductive parameters of male and female offspring up to 300 mg/kg (7-fold the human AUC 0-24 at the MRHD).
8.2Lactation Risk Summary There are no data on the presence of sepiapterin in either human or animal milk, the effects on the breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for SEPHIENCE and any potential adverse effects on the breastfed infant from SEPHIENCE or from the underlying maternal condition.
8.4Pediatric Use The safety and effectiveness of SEPHIENCE have been established in pediatric patients 1 month of age and older. Use of SEPHIENCE for this indication is supported by evidence from one adequate and well-controlled… [Excerpted — this section continues on DailyMed.]
🤰 Pregnancy ▾
8.1Pregnancy Risk Summary Available data on the use of SEPHIENCE during pregnancy are insufficient to evaluate for a drug-associated risk of major birth defects, miscarriage, or other adverse maternal or fetal outcomes. Uncontrolled blood Phe concentrations before and during pregnancy are associated with an increased risk of adverse pregnancy outcomes and fetal adverse effects ( see Clinical Considerations ) . In animal reproduction studies, oral administration of sepiapterin to pregnant rats and rabbits during organogenesis at dose exposures up to 9- and 6- times the human exposure at the maximum recommended human dose (MRHD) of 60 mg/kg, respectively, resulted in no adverse developmental effects ( see Data ).
All pregnancies have a background risk of birth defects, loss, or other adverse outcomes. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. The background risk of major birth defects and miscarriage in pregnant women with PKU who maintain blood Phe concentrations greater than 600 micromol/L during pregnancy is greater than the corresponding background risk for pregnant women without PKU.
Clinical Considerations Disease-Associated Maternal and/or Embryofetal Risk Uncontrolled blood Phe concentrations before and during pregnancy are associated with an increased risk of adverse outcomes and fetal adverse effects ( see Data ). To reduce the risk of hyperphenylalaninemia-induced fetal adverse effects, blood Phe concentrations should be maintained between 120 and 360 micromol/L during pregnancy and during the 3 months before conception [see Dosage and Administration ( 2.2 )] . Data Human Data Available data from the Maternal Phenylketonuria Collaborative Study on 468 pregnancies and 331 live births in pregnant women with PKU demonstrated that uncontrolled Phe concentrations above 600 micromol/L are associated with an increased risk for major birth defects (including microcephaly, major cardiac malformations), intrauterine fetal growth retardation, and future intellectual disability with low IQ.
Animal Data In an embryo-fetal development study in rats, SEPHIENCE was administered at dose levels of 100, 300, or 1,000 mg/kg/day via oral gavage to pregnant rats during the period of organogenesis from gestation day (GD) 7 to GD 17. There were no maternal or embryo-fetal developmental toxicities noted at doses up to 1,000 mg/kg/day (9-fold the human AUC 0-24 at the MRHD). In an embryo-fetal development study in pregnant rabbits, SEPHIENCE was administered at dose levels of 100, 300, or 1,000 mg/kg/day via oral gavage to pregnant rabbits during the period of organogenesis from gestation day (GD) 7 to GD 19.
There were no maternal or embryo-fetal developmental toxicities at doses up to 1,000 mg/kg/day (6-fold the human AUC 0-24 at the MRHD). In the pre- and post-natal development study in rats, SEPHIENCE was administered at dose levels of 30, 100, and 300 mg/kg/day via oral gavage once daily to pregnant rats from GD 6 to lactation day 20. SEPHIENCE did not induce effects on maternal reproductive function or on developmental and reproductive parameters of male and female offspring up to 300 mg/kg (7-fold the human AUC 0-24 at the MRHD).
🧒 Pediatric Use ▾
8.4Pediatric Use The safety and effectiveness of SEPHIENCE have been established in pediatric patients 1 month of age and older. Use of SEPHIENCE for this indication is supported by evidence from one adequate and well-controlled trial in 63 pediatric patients with PKU aged 1 to <17 years old (Trial 1) and additional safety and efficacy information obtained from an ongoing open label trial in adult and pediatric patients with PKU (Trial 2) [see Clinical Studies ( 14.1 )] . In Trial 1 Part 2, hypophenylalaninemia was seen in 5.4% (2/37) of sepiapterin-treated pediatric patients and in no adult patients.
Some pediatric patients in Trial 2 had multiple low blood Phe levels [see Adverse Reactions ( 6.1 )] . The safety and effectiveness of SEPHIENCE for treatment of PKU have not been established in pediatric patients younger than 1 month of age.
🧓 Geriatric Use ▾
8.5Geriatric Use Clinical studies of SEPHIENCE did not include patients 65 years of age and older to determine if they respond differently from younger adult patients.
🧬 Clinical Pharmacology ▾
12 CLINICAL PHARMACOLOGY
12.1Mechanism of Action SEPHIENCE is a precursor of the enzymatic co-factor tetrahydrobiopterin (BH 4 ) which activates PAH.
12.2Pharmacodynamics Cardiac Electrophysiology At the recommended SEPHIENCE dose of 60 mg/kg orally once daily, clinically significant QTc interval prolongation was not observed.
12.3Pharmacokinetics Following oral administration, sepiapterin reached the maximum concentration in plasma at 2 hours post-treatment and converted to pharmacologically active metabolite BH 4 with the exposures of sepiapterin generally less than 2% of those of BH 4 ( Table 5 ). There was no accumulation for BH 4 following repeated once daily dose up to 60 mg/kg administered in adult healthy volunteers. When administered with a high-fat, high-calorie meal in adult healthy volunteers, BH 4 exposures increased less than dose proportionally (with slopes 0.87 and 0.84 for C max and AUC 0-last , respectively) in the dose range 5 to 20 mg/kg and less than dose proportionally (with slopes 0.1957 and 0.3189 for C max and AUC 0-last , respectively) in the dose range 20 to 60 mg/kg.
The pharmacokinetics of sepiapterin and its active metabolite BH 4 following oral administration of sepiapterin at 60 mg/kg with food in adult patients with PKU are summarized in Table 5 . Table 5: Summary of Pharmacokinetic Parameters of Sepiapterin and BH 4 Following Oral Administration of Sepiapterin at 60 mg/kg with Food a in Adult Patients (Age ≥17 Years) with PKU a Sepiapterin was administered with dietary Phe restriction. NC: not calculated.
Sepiapterin plasma concentration quickly declines to below lower limit of quantitation by 12 hours post dose and AUCinf were not estimable for the majority of patients. Analyte T max (hr) Median (Range) C max (ng/mL) Mean (SD) AUC inf ng*hr/mL) Mean (SD) AUC 24 (ng*hr/mL) Mean (SD) Sepiapterin N Results 29 2 (0.5, 6.1) 29 2.9 (1.6) NC 27 19.9 (18.3) BH 4 N Results 30 4 (2, 8) 30 432 (177) 19 3,626 (1267) 30 3,618 (1,699) Absorption The time to maximum plasma concentration (T max ) of sepiapterin is approximately 1 to 3 hours after oral administration.
Plasma sepiapterin is rapidly metabolized to BH 4 and peak BH 4 concentrations are achieved approximately 4 hours after the oral administration of sepiapterin. Effect of Food Administration of SEPHIENCE with food results in increased exposure to sepiapterin and BH 4 . When SEPHIENCE was administered at 20 or 60 mg/kg daily with a low-fat meal in healthy adult subjects, BH 4 exposures were 169% to 172% higher for C max and 162% to 173% higher for AUC 0-24h compared to administration under fasted conditions.
When sepiapterin at 20 or 60 mg/kg was administered with a high-fat, high-calorie meal, BH 4 exposures were 221% to 226% higher for C max and 251% to 284% higher for AUC 0-24h compared to administration under fasted conditions [see Dosage and Administration ( 2.2 )] . Distribution Sepiapterin mean human plasma protein binding was 15.4% in the presence of 0.1% dithiothreitol (DTT) in the concentration range of 0.1 to 10 μM. BH 4 mean human plasma protein binding was between 24.1% and 41.3% in the concentration range 2 to 15 μM in the presence of 0.5% β-mercaptoethanol.
Sepiapterin apparent volume of distribution could not be estimated reliably as sepiapterin is converted to BH 4 post oral administration and plasma concentration declines to below lower limit of quantitation generally by 12 hours postdose. BH 4 apparent volume of distribution is 11433 (5790) L in adult patients with PKU. Increase of BH 4 in cerebrospinal fluid was detected after oral administration of sepiapterin 60 mg/kg QD for 7 days in healthy adult subjects.
Elimination Following oral administration, sepiapterin is quickly absorbed and converted to BH 4 . Sepiapterin plasma concentration is remarkably lower than BH 4 and declines rapidly to below the limit of quantitation generally by 12 hours post dose. The terminal half-life of BH 4 is approximately 5 hours and the appar… [Excerpted — this section continues on DailyMed.]
🧬 Mechanism of Action ▾
12.1Mechanism of Action SEPHIENCE is a precursor of the enzymatic co-factor tetrahydrobiopterin (BH 4 ) which activates PAH.
📦 How Supplied / Storage and Handling ▾
16 HOW 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: NDC 52856-201-03 Carton of 30-unit dose packets NDC 52856-201-01 Single unit dose packet 1,000 mg sepiapterin per packet: NDC 52856-301-03 Carton of 30-unit dose packets NDC 52856-301-01 Single unit dose packet Storage and Handling Store SEPHIENCE at room temperature between 20°C to 25°C (68°F to 77°F); excursion permitted between 15°C to 30°C (59°F to 86°F) [see USP Controlled Room Temperature].
Store SEPHIENCE liquid or soft food mixture either refrigerated or at controlled room temperature [see Dosage and Administration ( 2.4 )] .
📦 Storage and Handling ▾
Storage and Handling Store SEPHIENCE at room temperature between 20°C to 25°C (68°F to 77°F); excursion permitted between 15°C to 30°C (59°F to 86°F) [see USP Controlled Room Temperature]. Store SEPHIENCE liquid or soft food mixture either refrigerated or at controlled room temperature [see Dosage and Administration ( 2.4 )] .
📋 Description ▾
11 DESCRIPTION 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 (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one. The molecular formula is C9H11N5O3 and the molecular weight is 237.22 g/mol. The structural formula 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. Chemical Structure
💬 Information for Patients ▾
17 PATIENT 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 [see Drug Interactions ( 7 ) and Clinical Pharmacology ( 12.3 )] .
Feces Discoloration Advise patients that SEPHIENCE may cause feces to have a yellow or orange discoloration. Manufactured by Allphamed PHARBIL Arzneimittel GmbH Hildebrand str. 12, 37081 Gottingen, Germany (DEU) Manufactured for: PTC Therapeutics, Inc.
Warren, NJ 07059
🧬 Pharmacokinetics ▾
12.3Pharmacokinetics Following oral administration, sepiapterin reached the maximum concentration in plasma at 2 hours post-treatment and converted to pharmacologically active metabolite BH 4 with the exposures of sepiapterin generally less than 2% of those of BH 4 ( Table 5 ). There was no accumulation for BH 4 following repeated once daily dose up to 60 mg/kg administered in adult healthy volunteers. When administered with a high-fat, high-calorie meal in adult healthy volunteers, BH 4 exposures increased less than dose proportionally (with slopes 0.87 and 0.84 for C max and AUC 0-last , respectively) in the dose range 5 to 20 mg/kg and less than dose proportionally (with slopes 0.1957 and 0.3189 for C max and AUC 0-last , respectively) in the dose range 20 to 60 mg/kg.
The pharmacokinetics of sepiapterin and its active metabolite BH 4 following oral administration of sepiapterin at 60 mg/kg with food in adult patients with PKU are summarized in Table 5 . Table 5: Summary of Pharmacokinetic Parameters of Sepiapterin and BH 4 Following Oral Administration of Sepiapterin at 60 mg/kg with Food a in Adult Patients (Age ≥17 Years) with PKU a Sepiapterin was administered with dietary Phe restriction. NC: not calculated.
Sepiapterin plasma concentration quickly declines to below lower limit of quantitation by 12 hours post dose and AUCinf were not estimable for the majority of patients. Analyte T max (hr) Median (Range) C max (ng/mL) Mean (SD) AUC inf ng*hr/mL) Mean (SD) AUC 24 (ng*hr/mL) Mean (SD) Sepiapterin N Results 29 2 (0.5, 6.1) 29 2.9 (1.6) NC 27 19.9 (18.3) BH 4 N Results 30 4 (2, 8) 30 432 (177) 19 3,626 (1267) 30 3,618 (1,699) Absorption The time to maximum plasma concentration (T max ) of sepiapterin is approximately 1 to 3 hours after oral administration.
Plasma sepiapterin is rapidly metabolized to BH 4 and peak BH 4 concentrations are achieved approximately 4 hours after the oral administration of sepiapterin. Effect of Food Administration of SEPHIENCE with food results in increased exposure to sepiapterin and BH 4 . When SEPHIENCE was administered at 20 or 60 mg/kg daily with a low-fat meal in healthy adult subjects, BH 4 exposures were 169% to 172% higher for C max and 162% to 173% higher for AUC 0-24h compared to administration under fasted conditions.
When sepiapterin at 20 or 60 mg/kg was administered with a high-fat, high-calorie meal, BH 4 exposures were 221% to 226% higher for C max and 251% to 284% higher for AUC 0-24h compared to administration under fasted conditions [see Dosage and Administration ( 2.2 )] . Distribution Sepiapterin mean human plasma protein binding was 15.4% in the presence of 0.1% dithiothreitol (DTT) in the concentration range of 0.1 to 10 μM. BH 4 mean human plasma protein binding was between 24.1% and 41.3% in the concentration range 2 to 15 μM in the presence of 0.5% β-mercaptoethanol.
Sepiapterin apparent volume of distribution could not be estimated reliably as sepiapterin is converted to BH 4 post oral administration and plasma concentration declines to below lower limit of quantitation generally by 12 hours postdose. BH 4 apparent volume of distribution is 11433 (5790) L in adult patients with PKU. Increase of BH 4 in cerebrospinal fluid was detected after oral administration of sepiapterin 60 mg/kg QD for 7 days in healthy adult subjects.
Elimination Following oral administration, sepiapterin is quickly absorbed and converted to BH 4 . Sepiapterin plasma concentration is remarkably lower than BH 4 and declines rapidly to below the limit of quantitation generally by 12 hours post dose. The terminal half-life of BH 4 is approximately 5 hours and the apparent clearance is 1498 (848) L/h in adult patients with PKU.
Metabolism Sepiapterin is metabolized by SR/carbonyl reductase (CR) and DHFR in a 2-step unidirectional process to form pharmacologically active metabolite BH 4 . BH 4 is further metabolized non-enzymatically or enzymatically mediated by aromatic amino acid hydroxylases,… [Excerpted — this section continues on DailyMed.]
🧬 Pharmacodynamics ▾
12.2Pharmacodynamics Cardiac Electrophysiology At the recommended SEPHIENCE dose of 60 mg/kg orally once daily, clinically significant QTc interval prolongation was not observed.
🔬 Clinical Studies ▾
14 CLINICAL STUDIES
14.1Clinical Studies in PKU Trial 1 (NCT05099640) was a two-part trial in adult and pediatric patients who had a diagnosis of PKU with hyperphenylalaninemia with at least 2 blood Phe measurements ≥600 μmol/L. Patients in the trial were 54% male, 90% White, 5% American Indian or Alaska Native, and 4% Other by race; 16% were identified as Hispanic or Latino. The mean age of the trial patients was 17 years (range: 1 to 61 years).
At trial baseline, 8% of patients had blood Phe levels at <360 μmol/L, 36% at 360-600 μmol/L, 48% at 600-1200 μmol/L, and 8% at >1200 μmol/L. In Part 1, 157 patients received open-label treatment with SEPHIENCE: 7.5 mg/kg orally in patients 0 to <6 months of age, 15 mg/kg in patients 6 to <12 months of age, 30 mg/kg in patients 12 months to <2 years of age, or 60 mg/kg in patients ≥2 years of age per day for 14 days. In Part 1, 66% of PKU patients showed a biochemical response to SEPHIENCE with a >30% or greater reduction in Phe level.
In Part 2, after the 2-week washout period from Part 1, 98 patients aged 2 years and older who demonstrated a ≥30% reduction in blood Phe levels to SEPHIENCE treatment in Part 1 were randomized in a double-blind fashion to either SEPHIENCE 20 mg/kg daily for Weeks 1 and 2, 40 mg/kg daily for Weeks 3 and 4, 60 mg/kg daily for Weeks 5 and 6 (N=49), or placebo (N=49) for 6 weeks to assess efficacy. Twelve additional patients who had shown a 15% to 30% reduction in blood Phe during Part 1 were enrolled in Part 2 but were not included in the primary efficacy analysis.
In Part 2, the primary efficacy was assessed in patients who demonstrated a ≥30% reduction in blood Phe levels during Part 1 (N=98) by the mean change in blood Phe level from baseline to Weeks 5 and 6 in the SEPHIENCE-treated group as compared to the mean change in the placebo group, as shown in Table 7 . In Part 2 of Trial 1, 55 patients in the SEPHIENCE group and 54 patients in the placebo group completed the treatment period. One patient in the SEPHIENCE group discontinued treatment.
Table 7 displays a reduction in blood Phe level from baseline to Weeks 5 and 6 in Part 2 of Trial 1 for SEPHIENCE-treated patients relative to placebo. Figure 1 displays the mean blood Phe level over time by treatment group. Table 7: Change in Blood Phe Levels from Baseline at Weeks 5 and 6 For Patients Who Had a ≥30% Reduction in Blood Phe Levels During Part 1 of Trial 1 a Baseline is the average of Day -1 and Day 1 blood Phe levels in Part 2. b Blood Phe levels were based on average values during Weeks 5 and 6. † Adjusted mean and standard error, p-value <0.0001 for treatment difference were from a mixed model for repeated measures (MMRM) with change in blood Phe from baseline to post-baseline assessments as the response variable, and fixed effects for treatment, baseline blood Phe, baseline Phe stratum, visit and treatment-by-visit interaction.
SEPHIENCE (N=49) Placebo (N=49) Baseline Blood Phe Level a (μmol/L) Mean (±SD) 646.1 (253) 654 (261.5) Percentiles (25 th , 75 th ) 468.5, 769.5 466, 796 Weeks 5 and 6 b Mean (±SD) 236 (174.9) 637.9 (259.9) Percentiles (25 th , 75 th ) 127.7, 291 453, 776 Mean Change in Blood Phe From Baseline to Weeks 5 and 6 (μmol/L) Adjusted Mean (±SE) † -415.8 (24.1) -19.9 (24.2) Percentiles (25 th , 75 th ) -544, -296.7 -86.7,
63.7Mean Percent Change in Blood Phe From Baseline to Weeks 5 and 6 Mean (±SD) -62.8 (20.7) 1.4 (29.2) Percentiles (25 th , 75 th ) -76.9, -60.7 -12.4, 15 Treatment difference (95% CI) in adjusted mean -64.2 (-74.1, -54.4) Figure 1: Mean (±SD) Blood Phe Levels Over Time For Patients Who Had a ≥30% Reduction in Blood Phe Levels During Part 1 of Trial 1 (N=98) Supportive efficacy data were provided from Trial 2, which is an ongoing, multicenter, open-label trial in adult and pediatric patients with a clinical diagnosis of PKU with hyperphenylalaninemia.
At the data cutoff date, 169 patients, including 65 adult and 104 pediatric patients (median age: 14 years… [Excerpted — this section continues on DailyMed.]
🧪 Nonclinical Toxicology ▾
13 NONCLINICAL TOXICOLOGY
13.1Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenesis In a 6-month carcinogenicity study in Tg.rasH2 mice, sepiapterin did not increase the incidence of tumors in male or female transgenic mice at approximately 12- and 16-fold, respectively, the human exposure (AUC 0-24h ) at the MRHD. Mutagenesis Based on the weight of evidence, SEPHIENCE is not genotoxic. SEPHIENCE was negative in the Ames assay.
SEPHIENCE was positive in an in vitro chromosomal aberration assay without metabolic activation but not with metabolic activation. SEPHIENCE was negative in the in vivo (micronucleus and comet) assays in rats. Impairment of Fertility SEPHIENCE was found to have no effect on fertility and reproductive function of male and female rats when given prior to and throughout mating in male and female rats and continuing to gestation day (GD) 7 in females at oral doses up to 300 mg/kg/day (approximately 7.5-fold the plasma exposure (AUC) at MRHD).
13.2Animal Toxicology and/or Pharmacology Repeated administration of SEPHIENCE in rats for 26 weeks at doses greater than 100 mg/kg/day (approximately 3-fold the plasma exposure (AUC) at MRHD) caused renal toxicity, including renal tubular degeneration associated with crystal formation. However, renal toxicity was not noted in monkeys following repeated administration of SEPHIENCE for 39 weeks up to 300 mg/kg/day (approximately 7-fold the plasma exposure (AUC) at MRHD).
📄 Carcinogenesis, Mutagenesis, Impairment of Fertility ▾
13.1Carcinogenesis, Mutagenesis, Impairment of Fertility Carcinogenesis In a 6-month carcinogenicity study in Tg.rasH2 mice, sepiapterin did not increase the incidence of tumors in male or female transgenic mice at approximately 12- and 16-fold, respectively, the human exposure (AUC 0-24h ) at the MRHD. Mutagenesis Based on the weight of evidence, SEPHIENCE is not genotoxic. SEPHIENCE was negative in the Ames assay.
SEPHIENCE was positive in an in vitro chromosomal aberration assay without metabolic activation but not with metabolic activation. SEPHIENCE was negative in the in vivo (micronucleus and comet) assays in rats. Impairment of Fertility SEPHIENCE was found to have no effect on fertility and reproductive function of male and female rats when given prior to and throughout mating in male and female rats and continuing to gestation day (GD) 7 in females at oral doses up to 300 mg/kg/day (approximately 7.5-fold the plasma exposure (AUC) at MRHD).
📄 Patient Package Insert ▾
This Patient Information has been approved by the U.S. Food and Drug Administration. Issued: 07/2025 PATIENT INFORMATION SEPHIENCE (seh-FIGH-ence) (sepiapterin) oral powder What is SEPHIENCE?
SEPHIENCE is a prescription medicine used to lower blood levels of phenylalanine (Phe) in adults and children 1 month of age and older with a certain type of Phenylketonuria (PKU). SEPHIENCE is used along with a Phe-restricted diet. It is not known if SEPHIENCE is safe and effective in children younger than 1 month of age.
Before taking or giving SEPHIENCE, tell your healthcare provider about all of your medical conditions, including if you: are pregnant or plan to become pregnant. It is not known if SEPHIENCE will harm your unborn baby. are breastfeeding or plan to breastfeed. It is not known if SEPHIENCE passes into your breast milk.
Talk to your healthcare provider about the best way to feed your baby if you take SEPHIENCE. Tell your healthcare provider about all medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. SEPHIENCE and other medicines may affect each other causing side effects.
SEPHIENCE may affect the way other medicines work, and other medicines may affect how SEPHIENCE works. Especially tell your healthcare provider if you take: a medicine that contains levodopa an antifolate medicine another medicine also used to lower Phe medicines used to treat erectile dysfunction (phosphodiesterase 5 inhibitors or PDE5) Know the medicines you take. Keep a list of them with you to show your healthcare provider and pharmacist when you get a new medicine.
How should I take or give SEPHIENCE? Take or give SEPHIENCE exactly as your healthcare provider tells you. Your healthcare provider will tell you how much SEPHIENCE to take or give.
Your healthcare provider may change your or your child’s dose of SEPHIENCE depending on your weight and age. Take or give SEPHIENCE 1 time each day with food. You must take or give more food after SEPHIENCE dose.
SEPHIENCE comes as a packet containing oral powder. For SEPHIENCE doses less than 1,000 mg: mix SEPHIENCE oral powder with water or apple juice before taking or giving. For SEPHIENCE doses 1,000 mg or greater SEPHIENCE oral powder can be mixed with water, apple juice, strawberry jam or applesauce before taking or giving.
See the detailed Instructions for Use that comes with SEPHIENCE for information about the correct way to prepare, take, or give a dose of SEPHIENCE oral powder. If you or your child miss a dose of SEPHIENCE, take or give it as soon as possible. Do not take 2 doses in a day.
Continue with your normal dosing schedule the following day. During treatment with SEPHIENCE, you or your child will have regular check-ups with your healthcare provider. Your healthcare provider will do blood tests before and during your treatment with SEPHIENCE to check your blood Phe levels.
Your healthcare provider may change your dose, temporarily stop, or permanently stop treatment with SEPHIENCE if you have certain side effects Your healthcare provider may change your diet during treatment. Follow your healthcare provider’s instructions and do not make any changes to your diet without first talking to your healthcare provider. What are the possible side effects of SEPHIENCE?
SEPHIENCE may cause serious side effects including: increased bleeding problems. SEPHIENCE may increase your risk of bleeding. Tell your healthcare provider if you develop any symptoms of bleeding including: bruising or red or purple skin marks bleeding longer than usual or that does not stop menstrual bleeding that is heavier than normal low blood Phe (hypophenylalaninemia).
Low blood Phe levels is common during treatment with SEPHIENCE and can be severe. The most common side effects of SEPHIENCE include: diarrhea, headache, stomach (abdominal) pain, yellow or orange stool (feces), and throat pain. Tell your healthcare provider if you have any side effect that bothers you or that… [Excerpted — this section continues on DailyMed.]
📖 Instructions for Use ▾
INSTRUCTIONS FOR USE SEPHIENCE (seh-FIGH-ence) (sepiapterin) oral powder This Instructions for Use contains information on how to prepare, take, or give SEPHIENCE. Ask your healthcare provider or pharmacist if you have any questions on how to prepare, take, or give SEPHIENCE. The preparation steps differ based on the prescribed dose.
Follow the steps specific to the dose your healthcare provider has prescribed. See the section called: SEPHIENCE Doses Less Than 1,000 mg or Instructions for SEPHIENCE Doses 1,000 mg or Greater Each SEPHIENCE carton contains: 30 SEPHIENCE 250 mg dose packets or 30 SEPHIENCE 1,000 mg dose packets Important Information you need to know before taking or giving SEPHIENCE: SEPHIENCE comes as a packet containing powder. Take or give SEPHIENCE exactly as your healthcare provider tells you.
Be sure that you know what dose of SEPHIENCE your healthcare provider has prescribed. Your healthcare provider will prescribe SEPHIENCE 250 mg packet(s), SEPHIENCE 1,000 mg packet(s), or both types of packets to prepare your dose. Your healthcare provider may change your dose of SEPHIENCE depending on how you respond to treatment and based on your weight and age.
Take or give SEPHIENCE 1 time each day with food. You must take or give more food after each SEPHIENCE dose. Do not use SEPHIENCE oral powder packet(s) after the expiration date on the powder packet(s) and box.
The expiration date is the last day of the expiration month. For SEPHIENCE doses less than 1,000 mg: SEPHIENCE oral powder is to be mixed with water or apple juice before taking or giving. Follow the section called Instructions for SEPHIENCE doses less than 1,000 mg .
For SEPHIENCE doses 1,000 mg or greater: SEPHIENCE oral powder can be mixed with water, apple juice, strawberry jam, or applesauce before taking or giving. Follow the section called Instructions for SEPHIENCE Doses 1,000 mg or Greater . Storing SEPHIENCE: Store SEPHIENCE oral powder packet(s) at room temperature between 68°F to 77°F (20°C to 25°C).
For SEPHIENCE oral powder that is mixed with water, apple juice, strawberry jam, or applesauce: If the SEPHIENCE mixture is not taken or given right away, cover and store the mixture at room temperature between 68°F to 77°F (20°C to 25°C) for up to 6 hours or in the refrigerator between 36°F to 46°F (2°C to 8°C) for up to 24 hours. If the stored SEPHIENCE mixture was prepared with water or apple juice , stir the mixture for 30 seconds or more before taking or giving the dose. If the stored SEPHIENCE mixture was prepared with strawberry jam or applesauce , stir the mixture for 60 seconds or more before taking or giving the dose.
Throw away (discard) unused SEPHIENCE mixture after 6 hours at room temperature or after 24 hours if refrigerated. See Throwing away (disposing of) SEPHIENCE . Keep SEPHIENCE and all medicines out of the reach of children.
Preparing to mix SEPHIENCE oral powder: Instructions for SEPHIENCE Doses Less Than 1,000 mg: Your healthcare provider or pharmacist will tell you the amount of: Water or apple juice needed to mix 1 dose of SEPHIENCE. SEPHIENCE mixture (after mixed with water or apple juice) to give in milliliters (mL). You may need to measure a smaller amount of mixture than you prepared to take or give the correct prescribed dose of SEPHIENCE.
Supplies needed to mix and give SEPHIENCE doses less than 1,000 mg (see Figure A ): The number of SEPHIENCE 250 mg oral powder packet(s) needed for 1 dose, or one SEPHIENCE 1,000 mg packet(s) Supplies not included with the SEPHIENCE oral powder packet carton(s): 1 small cup of water or apple juice 1 container for mixing 1 small spoon a 10 mL oral dosing syringe or recommended oral dosing syringe scissors (optional) Ask your pharmacist for a container for mixing SEPHIENCE and an oral dosing syringe if you do not have these supplies.
Step 1: Find a clean, flat work surface. Place the following items on your clean, flat work surface: prescribed number of SEPHIENCE oral powder packet… [Excerpted — this section continues on DailyMed.]
📄 Package Label / Principal Display Panel ▾
Package Labeling:60468-006-03 Outer Label0 Inner Label0 Box
Package Labeling: 60468-007-03 Outer Label2 Inner Label2
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