Sephience
Sephience (sepiapterin) is a phenylalanine hydroxylase (PAH) activator that lowers high blood phenylalanine (Phe) levels in adults and children ages 1 month and older with phenylketonuria (PKU). It’s used along with a Phe-restricted diet. The medication comes as powder packets that you mix with liquid or soft food and take by mouth once a day with food. Your dose depends on your age, weight, and Phe levels. Common side effects include diarrhea, headache, and stomach pain.
What is Sephience (sepiapterin)?
What is Sephience (sepiapterin) used for?
- Treat high blood phenylalanine (Phe) levels in adults and children ages one month and older with phenylketonuria (PKU), along with a Phe-restricted diet
How Sephience (sepiapterin) works
Sephience (sepiapterin) is a phenylalanine hydroxylase (PAH) activator. Phenylalanine (Phe) is an amino acid found in protein-containing foods. If you have PKU, a protein called PAH doesn’t work well enough to break down Phe, causing it to build up in your blood.
Sephience (sepiapterin) helps PAH work better, which lowers your Phe levels and keeps them in a safer range.
Drug facts
| Common Brands | Sephience |
|---|---|
| Drug Class | Phenylalanine hydroxylase activator |
| Controlled Substance Classification | Not a controlled medication |
| Generic Status | No lower-cost generic available |
| Availability | Prescription only |
Side effects of Sephience (sepiapterin)
The following side effects may get better over time as your body gets used to the medication. Let your healthcare provider know immediately if you continue to experience these symptoms or if they worsen over time.
Common Side Effects
- Diarrhea (7%)
- Headache (7%)
- Stomach pain (5%)
- Low blood Phe (4%)
- Changes in stool color (4%)
- Mouth or throat pain (4%)
Less Common Side Effects
- Bleeding or bruising
Sephience (sepiapterin) serious side effects
Contact your healthcare provider immediately if you experience any of the following.
- Bleeding: easy bruising, red or purple marks on the skin, bleeding that lasts longer than usual, heavy menstrual bleeding
- Low blood Phe: slow growth, poor weight gain, tiredness, lack of energy, rash, hair thinning, diarrhea
The following Sephience (sepiapterin) side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Bleeding—bloody or black, tar-like stools, vomiting blood or brown material that looks like coffee grounds, red or dark brown urine, small red or purple spots on skin, unusual bruising or bleeding
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
- Dark yellow or orange stools
- Diarrhea
- Headache
- Sore throat
- Stomach pain
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Pros and cons of Sephience (sepiapterin)
Pros
- Taken by mouth once a day
- Approved for adults and children as young as 1 month old
- Powder packets can be mixed with liquid or soft food
Cons
- Requires a Phe-restricted diet and regular blood tests
- Commonly causes diarrhea, headache, and abdominal pain
- Raises the risk of bleeding and low blood Phe levels, especially in children
Pharmacist tips for Sephience (sepiapterin)
- Take Sephience (sepiapterin) once a day with food to help your body absorb it. Eat more food after your dose as directed.
- Keep your Phe-restricted diet consistent while your response with Sephience (sepiapterin) is being checked. Changing how much protein or Phe you eat can affect your blood Phe level and make it harder to tell how well treatment is working.
- Keep all blood Phe test appointments while taking Sephience (sepiapterin). These tests help your care team adjust your dose or diet and find Phe levels that are too high or too low.
- Your care team will check your blood Phe level within 2 weeks after you start Sephience (sepiapterin). If it doesn’t drop after 2 weeks at the maximum dose of 60 mg/kg, your prescriber will likely stop treatment.
- If you miss a dose of Sephience (sepiapterin), take it as soon as you remember. Don’t take 2 doses on the same day, and return to your regular schedule the next day.
- Tell your prescriber and pharmacist about all medication and supplements you take with Sephience (sepiapterin). Some medications, like trimethoprim and methotrexate, can make treatment work less well and should generally be avoided.
- Tell your care team if you take levodopa or sildenafil with Sephience (sepiapterin). Levodopa can raise the risk of seizures, overstimulation, or irritability, while sildenafil and similar medications can lower your blood pressure and cause dizziness or fainting.
- Watch for unusual bleeding while taking Sephience (sepiapterin). Tell your prescriber about easy bruising, red or purple marks, bleeding that lasts longer than usual, or heavier menstrual bleeding. Get medical help right away if bleeding is heavy or won’t stop.
- Sephience (sepiapterin) can make your stool look yellow or orange. This color change is a known side effect.
- Pregnancy and Breastfeeding: Tell your prescriber if you’re pregnant, planning to become pregnant, or breastfeeding while taking Sephience (sepiapterin). There isn’t enough information to know how the medication affects pregnancy or a breastfed baby.
- Storage: Keep unmixed Sephience (sepiapterin) packets at room temperature. After mixing a dose, you can keep it covered at room temperature for up to 6 hours or refrigerated for up to 24 hours. Throw away any mixture after these time limits.
How to take Sephience (sepiapterin)
- For doses under 1,000 mg: mix the powder with the instructed amount of water or apple juice.
- For doses 1,000 mg or more: mix the packets with the instructed amount of water, apple juice, strawberry jam, or applesauce.
- Stir liquid mixtures for at least 30 seconds and soft-food mixtures for at least 60 seconds.
- Take the full mixture right away after preparing it. If powder remains in the container or syringe, rinse it with water or apple juice and take the rinse right away.
Risks and warnings for Sephience (sepiapterin)
Sephience (sepiapterin) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Increased bleeding
Sephience (sepiapterin) can raise your risk of bleeding. Tell your prescriber if you notice easy bruising, red or purple marks on your skin, frequent nosebleeds, bleeding gums, bleeding that lasts longer than usual, or heavier menstrual bleeding. Get medical help right away if bleeding is heavy or won’t stop. Your prescriber might pause Sephience (sepiapterin) if you have active bleeding.
Low blood Phe levels
- Risk factors: Children
Sephience (sepiapterin) can lower your blood Phe level too much. This has been reported in children with PKU. If low Phe levels continue for a long time, they can affect nutrition and growth. Your care team will adjust your medication dose, dietary protein, and Phe intake to keep your blood Phe in a safe range.
Keep all blood Phe tests and diet appointments while taking Sephience (sepiapterin). Children might need testing more often because their Phe levels can change as they grow. If your child takes this medication, tell their care team about poor appetite, low energy, trouble gaining weight, or changes in growth or development. The prescriber can lower the dose or adjust the diet if needed.
Interaction with levodopa
- Risk factors: Taking levodopa | Brain or nervous system condition
Taking Sephience (sepiapterin) with levodopa, a medication commonly used to manage symptoms of Parkinson’s disease, might cause or worsen seizures, overstimulation, or irritability. These problems were reported in people who already had a brain or nervous system condition. Tell your prescriber and pharmacist if you take levodopa, including combination medications such as Sinemet or Rytary, so they can monitor you for changes in your mood, behavior, or nervous system.
Sephience (sepiapterin) dosage
Carton| Dosage | Quantity | Price as low as | Price per unit |
|---|---|---|---|
| 30 packets of 250mg | 3 cartons | $11,807.48 | $3,935.83 |
| 30 packets of 1000mg | 1 carton | $15,741.25 | $15,741.25 |
Typical dosage for Sephience (sepiapterin)
Your dose depends on your age and weight. Your prescriber might adjust it based on your Phe levels.
- Younger than 6 months: The typical starting dose is 7.5 mg/kg by mouth once a day with food.
- 6 months to younger than 1 year: The typical starting dose is 15 mg/kg by mouth once a day with food.
- 1 year to younger than 2 years: The typical starting dose is 30 mg/kg by mouth once a day with food.
- 2 years and older: The typical starting dose is 60 mg/kg by mouth once a day with food.
How much does Sephience (sepiapterin) cost?
$11,807.48
What are alternatives to Sephience (sepiapterin)?
Sephience (sepiapterin) images
