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

What is ZELVYSIA?

Zelvysia is a prescription medicine used to lower blood levels of phenylalanine (Phe), in adults and children one month of age and older with a certain type of Phenylketonuria (PKU). Zelvysia is used along with a Phe-restricted diet.

What should I tell my doctor before taking Zelvysia?

Before you take Zelvysia , tell your doctor about all your medical conditions, including if you:

are allergic to sapropterin dihydrochloride or any of the ingredients in Zelvysia . See the list of ingredients in Zelvysia at the end of this leaflet.
have poor nutrition or have loss of appetite.
are pregnant or plan to become pregnant.
are breastfeeding or plan to breastfeed. It is not known if Zelvysia passes into your breast milk. Talk to your doctor about the best way to feed your baby if you take Zelvysia .

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, herbal, and dietary supplements. Zelvysia and other medicines may interact with each other.

Especially tell your doctor if you take:

a medicine that contains levodopa
an antifolate medicine
sildenafil (Revatio, Viagra), tadalafil (Adcirca, Cialis), vardenafil (Staxyn, Levitra)

Tell your doctor if you are not sure if your medicine is one that is listed above.

Know the medicines you take. Keep a list of them to show your doctor and pharmacist when you get a new medicine.

How should I take ZELVYSIA ?

Take Zelvysia exactly as your doctor tells you. Your doctor should tell you how much Zelvysia to take and when to take it.
Your doctor may change your dose of Zelvysia depending on how you respond to treatment.
Take Zelvysia 1 time each day with a meal. It is best to take Zelvysia at the same time each day.
Sapropterin dihydrochloride comes as a powder for oral solution.
- Be sure that you know what dose of Zelvysia powder your doctor prescribed and whether you should use sapropterin dihydrochloride 100 mg packets, sapropterin dihydrochloride 500 mg packets, or both types of packets to prepare your dose.
- Open Zelvysia powder packets only when you are ready to use them.
- Zelvysia powder for oral solution should be dissolved in water or apple juice.You may also mix the powder for oral solution in a small amount of soft food, such as apple sauce or pudding before taking.
- See the detailed “Instructions for Use” that comes with Zelvysia for information about the correct way to dissolve and take a dose of Zelvysia powder for oral solution.

It is not possible to know if Zelvysia will work for you until you start taking Zelvysia . Your doctor will check your blood Phe levels when you start taking Zelvysia to see if the medicine is working.

During treatment with Zelvysia :
- Any change you make to your diet may affect your blood Phe level. Follow your doctor’s instructions carefully and do not make any changes to your dietary Phe intake without first talking with your doctor. Even if you take Zelvysia, if your Phe blood levels are not well controlled, you can develop severe neurologic problems.
- Your doctor should continue to monitor your blood Phe levels often during your treatment with Zelvysia to make sure that your blood Phe levels are not too high or too low.
- If you have a fever, or if you are sick, your blood Phe level may go up. Tell your doctor as soon as possible so they can change your dose of Zelvysia to help keep your blood Phe levels in the desired range.

If you forget to take your dose of Zelvysia, take it as soon as you remember that day. Do not take 2 doses in a day.
If you take too much Zelvysia, call your doctor for advice.

What are the possible side effects of ZELVYSIA ?

Zelvysia can cause serious side effects, including:

Severe allergic reactions.Stop taking Zelvysia and get medical help right away if you develop any of these symptoms of a severe allergic reaction:

wheezing or trouble breathing
coughing
feeling lightheaded or you faint

flushing
nausea
rash

Inflammation of the lining of the stomach (gastritis) or esophagus (esophagitis). Gastritis or esophagitis can happen with Zelvysia and may be severe. Call your doctor right away if you have any of these signs or symptoms:
- severe upper stomach-area (abdominal) discomfort or pain, nausea and vomiting
- blood in your vomit or stool
- black, tarry stools
- difficulty swallowing
- loss of appetite
- pain in the throat
Phe levels that are too low.Some children under the age of 7 years who take high doses of Zelvysia each day may experience low Phe levels.
Too much or constant activity (hyperactivity) can happen with Zelvysia. Tell your doctor if you have any signs of hyperactivity, including:
- fidgeting or moving around too much
- talking too much

The most common side effects of Zelvysia are:

headache
runny nose and nasal congestion
sore throat
diarrhea
vomiting
cough

Tell your doctor if you have any side effect that bothers you or that does not go away.

These are not all the possible side effects of Zelvysia . For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

How should I store ZELVYSIA ?

Store Zelvysia at room temperature between 68°F to 77°F (20°C to 25°C).
Protect from moisture.

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

General information about the safe and effective use of ZELVYSIA .

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

What are the ingredients in ZELVYSIA ?

Active ingredient:sapropterin dihydrochloride.

Zelvysia powder for oral solution inactive ingredients:ascorbic acid, mannitol, and potassium citrate.

Manufactured for:

Aucta Pharmaceuticals, Inc.

Piscataway, NJ 08854

For more information, go to www.Zelvysia.com or call 1-800-655-9902.

This Patient Information has been approved by the U.S. Food and Drug Administration

Revised: 01/2025

LB5405-01

INSTRUCTIONS FOR USE

Zelvysia (zel-VIS-ee-ah)

(sapropterin dihydrochloride)

powder for oral solution

Read this Instructions for Use before you start taking Zelvysia and each time you refill your prescription. There may be new information. This information does not take the place of talking with your healthcare provider about your treatment. Talk to your doctor if you have any questions about the right dose of Zelvysia to take or how to mix it.

Important information:

Zelvysia comes as a packet containing powder.
Take Zelvysia exactly as your doctor tells you. Your doctor should tell you how much Zelvysia to take and when to take it.
Your doctor may change your dose of Zelvysia depending on how you respond to treatment, or based on your baby’s weight.
If your baby weighs 22 pounds or less, follow the section called “ Instructions for giving Zelvysia powder for oral solution (Zelvysia 100 mg packets) to babies who weigh 22 pounds or less”.
Take Zelvysia 1 time each day with a meal. It is best to take Zelvysia at the same time each day.

Instructions for taking Zelvysia powder for oral solution:

For babies who weigh 22 pounds or less, see the section below called “ Instructions for giving Zelvysia powder for oral solution (Zelvysia 100 mg packets) to babies who weigh 22 pounds or less.”

Zelvysia powder for oral solution should be dissolved in water or apple juice. The powder for oral solution may also be mixed in a small amount of soft foods, such as apple sauce or pudding.

To dissolve Zelvysia powder for oral solution:

Be sure that you know what dose of Zelvysia your doctor has prescribed and whether you should use Zelvysia 100 mg packets, Zelvysia 500 mg packets, or both types of packets to prepare your dose.
Open the packet(s) of Zelvysia powder for oral solution by folding and tearing, or cutting at the dotted line in the upper right corner of the packet. Open the packet(s) only when you are ready to use them.
Empty the contents of the packet(s) into 4 ounces to 8 ounces (1/2 cup to 1 cup) of water or apple juice.
Drink within 30 minutes.

Instructions for giving Zelvysia powder for oral solution (Zelvysia 100 mg packets) to babies who weigh 22 pounds or less:

The dose of Zelvysia is based on body weight. This will change as your baby grows. Your doctor will tell you:
- the number of Zelvysia 100 mg packets needed for one dose
- the amount of water or apple juice needed to mix one dose of Zelvysia
- the amount of the mixture (powder and water or apple juice) you will need to give your baby his or her prescribed dose of medicine.
Give your baby the prescribed amount of mixture (powder and water or apple juice) within 30 minutes after mixing. If you are not able to give your baby’s dose within 30 minutes after mixing, pour the unused medicine into the trash. You will need to mix a new dose.

Supplies needed to mix and give your baby’s dose of Zelvysia powder for oral solution:

the number of Zelvysia 100 mg packets needed for one dose
a small cup of water or apple juice
one 30 mL medicine cup for mixing
small spoon or clean utensil for mixing
10 mL oral dosing syringe
scissors (optional)

Ask your pharmacist for a 30 mL medicine cup for mixing and an oral dosing syringe if you do not have these supplies.

Step 1:	Find a clean, flat work surface.
Step 2:	Place a small cup of water or apple juice, the oral dosing syringe, and an empty medicine cup on your clean, flat work surface (see Figure A).
Step 3:	Pour 5 mL or 10 mL of water or apple juice from the small cup into the medicine cup, as instructed by your doctor. Check to make sure that the amount of liquid lines up with the amount that your doctor tells you (see Figure B).
Step 4:	Check the label on the Zelvysia packet(s). If the packet is marked Zelvysia 100 mg, empty the entire contents of the Zelvysia packet into the medicine cup (see Figure C).
Step 5:	Stir the mixture with the small spoon or other clean utensil until all of the powder completely dissolves (see Figure D).
Step 6:	To give a dose of Zelvysia to your baby: Place the tip of the oral dosing syringe into the liquid inside the medicine cup. Pull back on the plunger and draw up the amount of the mixture prescribed by your doctor (see Figure E).
Step 7:	Take the oral dosing syringe out of the medicine cup. Carefully turn the oral dosing syringe so that the tip is pointing up. Check to make sure that the amount of medicine in the oral dosing syringe lines up with the amount of mixture prescribed by your doctor (see Figure F).
Step 8:	Place the tip of the oral dosing syringe into your baby’s mouth. Point the tip of the oral dosing syringe toward either cheek (see Figure G). Push on the plunger slowly, a small amount at a time, until all of the mixture in the oral dosing syringe is given.
Step 9:	Throw away any remaining mixture. Remove the plunger from the barrel of the oral dosing syringe. Wash the oral dosing syringe and medicine cup 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 medicine cup for the next use.