Patient information: uses, side effects, how to take - Sephience

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 does not go away.

These are not all of the possible side effects of SEPHIENCE.

Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

You may also report side effects to PTC Therapeutics, Inc. at 1-866-562-4620 or email at usmedinfo@ptcbio.com.

How should I store SEPHIENCE?

Store SEPHIENCE oral powder packets 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 refrigerate between 36°F to 46°F (2°C to 8°C) for no more than 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.

Keep SEPHIENCE and all medicines out of the reach of children.

General information about the safe and effective use of SEPHIENCE.

Medicines are sometimes prescribed for purposes other than those listed in a Patient Information leaflet. Do not use SEPHIENCE for a condition for which it was not prescribed. Do not give SEPHIENCE to other people, even if they have the same symptoms that you have. It may harm them. You can ask your pharmacist or healthcare provider for information about SEPHIENCE that is written for health professionals.

What are the ingredients in SEPHIENCE?

Active ingredients: sepiapterin

Inactive ingredients: colloidal silicon dioxide, croscarmellose sodium, isomalt, magnesium stearate, mannitol,

microcrystalline cellulose, sucralose, and xanthan gum.

Manufactured for:

PTC Therapeutics, Inc.

Warren, NJ 07059

SEPHIENCE is a registered trademark of PTC Therapeutics, Inc.

For more information, go to www.sephience.com or call 1-844-478-2227.

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 packets for your daily dose a small cup of water or apple juice container for mixing small spoon oral dosing syringe
Step 2: Check the label on the SEPHIENCE oral powder packet(s) to make sure you have the number of packets for the prescribed dose. Open the SEPHIENCE oral powder packet(s) by folding and tearing at the tear notch, or using scissors to cut the packet(s) open at the dotted line. Empty the entire contents of each SEPHIENCE oral powder packet(s) into the container for mixing (see Figure B ).
Step 3: Place the tip of the oral dosing syringe into the cup containing the water or apple juice. Pull back on the plunger and draw up the amount of water or apple juice needed to prepare the packet(s) for the prescribed dose. Use 9 mL of water or apple juice for each 250 mg packet. For example: If you are preparing 1 packet of SEPHIENCE, you will need to add 9 mL of water or apple juice. If you are preparing 2 packets of SEPHIENCE, you will need to add 18 mL of water or apple juice. If you are preparing 3 packets of SEPHIENCE, you will need to add 27 mL of water or apple juice. If you are preparing 4 packets of SEPHIENCE, you will need to add 36 mL of water or apple juice. If you are using one 1,000 mg packet to prepare the prescribed dose, you will need to add 36 mL of water or apple juice. Slowly add the water or apple juice to the container for mixing containing the SEPHIENCE oral powder. Repeat until the entire amount of water or apple juice that is needed to mix the number of SEPHIENCE oral powder packets has been added to the container for mixing (see Figure C ).
Step 4: Stir the SEPHIENCE oral powder mixed with water or apple juice well for 30 seconds or more with a small spoon until the SEPHIENCE mixture is free of lumps (see Figure D ). SEPHIENCE oral powder is not expected to dissolve completely. This is normal.
Step 5: Place the tip of the oral dosing syringe into the mixture inside the container used for mixing. Pull back on the plunger until the edge of the plunger lines up with the marking for the dose as instructed by your healthcare provider in mL (milliliters) (see Figure E ).
Step 6: Take the oral dosing syringe out of the container used for mixing. Carefully turn the oral dosing syringe so that the tip is pointing up. Check the syringe marking to make sure that the amount of SEPHIENCE mixture in the oral dosing syringe lines up with the prescribed SEPHIENCE dose in mL (milliliters) (see Figure F ). Your dose may be different than the dose shown in Figure F . Give SEPHIENCE mixture right away after it is drawn up into the syringe.
Step 7: Place the tip of the oral dosing syringe into your child’s mouth. Point the tip of the oral dosing syringe toward either cheek (see Figure G ). Push on the plunger slowly, until all the mixture in the oral dosing syringe is given. If the prescribed dose is more than 10 mL, repeat Step 5 to Step 7 until you give the total amount of prescribed dose in mL (milliliters) by your healthcare provider.
Step 8: If there is any mixture left in the oral dosing syringe, draw up more water or apple juice into the oral dosing syringe and give until no mixture is left in the oral dosing syringe.
Step 9: Throw away any SEPHIENCE mixture remaining in the container used for mixing in the household trash. See Throwing away (disposing of) SEPHIENCE. Remove the plunger from the barrel of the oral dosing syringe. Wash the oral dosing syringe and container used for mixing with warm water and air dry. When the oral dosing syringe is dry, put the plunger back into the barrel. Store the oral dosing syringe and container used for mixing for the next use.

Instructions for SEPHIENCE Doses 1,000 mg or Greater:
Supplies needed to mix and take or give SEPHIENCE doses 1,000 mg or greater (see Figure A): The number of SEPHIENCE 250 mg packet(s) or SEPHIENCE 1,000 mg packet(s) needed for 1 dose
Supplies not included with the SEPHIENCE oral powder packet carton(s): a small cup of water, apple juice, strawberry jam, or applesauce 1 tablespoon to measure the water, apple juice, strawberry jam, or applesauce additional small cup of water or apple juice to rinse the cup 1 container for mixing scissors (optional)
Your healthcare provider will tell you the number of packets, and the amount of water, apple juice, strawberry jam, or applesauce needed to mix 1 dose of SEPHIENCE per Table 1 below.

Table 1: Number of SEPHIENCE Packets to Prepare a SEPHIENCE Mixture for Doses of 1,000 mg or Greater

Find your prescribed number of packets:		Choose one of the following: Water Apple juice Strawberry jam Applesauce
Number of 1,000 mg packets	Number of 250 mg packets	Amount of water, apple juice, strawberry jam, or applesauce to mix the packet(s)
1	0	2 Tbsp or 30 mL
1	1	4 Tbsp or 60 mL
1	2	4 Tbsp or 60 mL
1	3	4 Tbsp or 60 mL
2	0	4 Tbsp or 60 mL
2	1	6 Tbsp or 90 mL
2	2	6 Tbsp or 90 mL
2	3	6 Tbsp or 90 mL
3	0	6 Tbsp or 90 mL
3	1	8 Tbsp or 120 mL
3	2	8 Tbsp or 120 mL
3	3	8 Tbsp or 120 mL
4	0	8 Tbsp or 120 mL
4	1	10 Tbsp or 150 mL
4	2	10 Tbsp or 150 mL
4	3	10 Tbsp or 150 mL
5	0	10 Tbsp or 150 mL
5	1	12 Tbsp or 180 mL
5	2	12 Tbsp or 180 mL
5	3	12 Tbsp or 180 mL
6	0	12 Tbsp or 180 mL

Ask your pharmacist for a container for mixing SEPHIENCE 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 packets a small cup of water, apple juice, strawberry jam, or applesauce additional small cup of water or apple juice to rinse the cup (see Step 6 below) tablespoon container for mixing
Step 2: Check the label on the SEPHIENCE oral powder packet(s) to make sure you have the number of packets for the prescribed dose. Open the SEPHIENCE oral powder packet(s) by folding and tearing at the tear notch or using scissors to cut the packet(s) open at the dotted line. Empty the entire contents of each SEPHIENCE oral powder packet(s) into the container for mixing (see Figure B ).
Step 3: Mix the SEPHIENCE oral powder packet(s) with water, apple juice, strawberry jam, or applesauce. See Table 1 above. Slowly add the water, apple juice, strawberry jam, or applesauce to the container for mixing containing the SEPHIENCE oral powder. Repeat until the entire amount of water, apple juice, strawberry jam, or applesauce that is needed to mix the number of SEPHIENCE oral powder packets has been added to the container for mixing (see Figure C ).
Step 4: If you used water or apple juice to mix the SEPHIENCE oral powder packet(s), stir the mixture well for 30 seconds or more with the tablespoon until the SEPHIENCE mixture is free of lumps (see Figure D ). SEPHIENCE oral powder is not expected to dissolve completely. or If you used strawberry jam or applesauce to mix the SEPHIENCE oral powder packet(s), stir the mixture well for 60 seconds or more with the tablespoon.
Step 5: Give or take all of the SEPHIENCE mixture from the container used for mixing.
Step 6: If any SEPHIENCE mixture remains in the container used for mixing, rinse the container with water or apple juice (at least 15 mL). Swallow the rinse right away. Repeat this step until no mixture remains in the container used for mixing.
Step 7: Wash the container used for mixing with warm water and air dry. Store the container used for mixing for the next use.

Throwing away (disposing of) SEPHIENCE:

Ask your pharmacist how to properly throw away (dispose of) SEPHIENCE oral powder packet(s) that are not needed or that have expired.

Manufactured for:

PTC Therapeutics, Inc.

Warren, NJ 07059

This Instructions for Use has been approved by the U.S. Food and Drug Administration.

Approved: 4/2026

Sephience (Sepiapterin): Uses, Side Effects and Dosage