Key takeaways
- Procysbi (cysteamine) is a prescription drug approved to treat nephropathic cystinosis in adults and some children. It does not cure the underlying genetic disorder, but it can ease symptoms and prevent further organ damage.
- Procysbi comes as delayed-release oral capsules and oral granules. It is typically taken twice per day and the dose depends on your body weight.
- Some examples of side effects of Procybsi include nausea and vomiting, abdominal pain, diarrhea, and bad breath.
Specifically, Procysbi is used in adults and children ages 1 year and older to treat nephropathic cystinosis..
To learn more about Procysbi’s uses, see the “Uses for Procysbi” section.
Procysbi basics
Procysbi contains the active ingredient cysteamine. It belongs to a group of drugs called cystine-depleting agents.
Procysbi is a brand-name medication. A generic version of the drug isn’t currently available.
Procysbi is used to treat nephropathic cystinosis in adults and children ages 1 year and older.
Nephropathic cystinosis is a rare, inherited disorder. It causes cystine (an amino acid) to build up in your body, which can cause serious problems. Nephropathic cystinosis can affect the thyroid gland, eyes, brain, heart, muscles, white blood cells, pancreas, and cause kidney damage.
Symptoms typically begin around 6 to 12 months of age and can include:
- frequent urination
- excessive thirst
- electrolyte imbalance
- weakened bones
- muscle spasms
- poor appetite and fussy eating
- vomiting
Procysbi works to treat nephropathic cystinosis by changing cystine into other substances inside your cells. By lowering the levels of cystine in your cells, it prevents crystals of cystine from forming. Procysbi helps reduce your symptoms and prevent or delay damage to organs, but it does not cure the disorder.
Like most drugs, Procysbi may cause mild to serious side effects. The following lists contain some of the more common side effects Procysbi may cause, but they don’t include all possible side effects.
Keep in mind that side effects of a drug can depend on:
- your age
- other health conditions you have
- other medications you take
Your doctor or pharmacist can tell you more about the potential side effects of Procysbi. They can also suggest ways to help reduce side effects.
Note: After the Food and Drug Administration (FDA) approves a drug, it tracks and reviews side effects of the medication. Sharing your experience helps make medications safer for everyone by giving doctors and researchers more information about how the drug works in real life. If you develop a side effect while taking Procysbi and want to tell the FDA about it, visit MedWatch or call 800-FDA-1088.
Mild side effects
Here’s a list of some of the mild side effects that Procysbi can cause. To learn about other mild side effects, talk with your doctor or pharmacist, or read Procysbi’s prescribing information.
Mild side effects of Procysbi that have been reported include:
- abdominal pain
- nausea and vomiting
- loss of appetite
- diarrhea
- stomach flu
- upper respiratory infection, such as the common cold
- pink eye
- ear infection
- fatigue (low energy)
- headache
- dizziness
- rash
- joint pain
- bad breath
- electrolyte imbalance
Mild side effects of many drugs may go away within a few days to a couple of weeks. But if they become bothersome, talk with your doctor or pharmacist.
Serious side effects
Serious side effects from Procysbi can occur, but they aren’t common. If you have serious side effects from Procysbi, call your doctor right away. If you think you’re having a medical emergency, you should call 911 or your local emergency number.
Serious side effects of Procysbi that have been reported include:
- fibrosing colonopathy (swelling and narrowing of the large intestine)
- brain and nervous system symptoms, such as seizures, drowsiness, or depression
- benign intracranial hypertension (increased pressure inside the skull)
- skin, bone, and joint problems, such as bone fractures, stretch marks, or joint hyperextension
- severe skin reactions, such as toxic epidermal necrolysis
- bleeding or ulcers in the stomach or intestine
- unusual changes in liver function tests
- leukopenia (low white blood cell count)
Allergic reactionFor some people, Procysbi can cause an allergic reaction.
In general, symptoms of an allergic reaction can be mild or serious.
What might helpIf you have mild symptoms of an allergic reaction, such as a mild rash, call your doctor right away. They may suggest a treatment to manage your symptoms and can determine whether you should continue Procysbi treatment.
If you have symptoms of a severe allergic reaction, such as swelling or difficulty breathing, call 911 or your local emergency number right away. These symptoms could be life threatening and require immediate medical care.
If your doctor confirms you’ve had a serious allergic reaction to Procysbi, they may have you switch to a different treatment.
The Procysbi dosage your doctor prescribes will depend on several factors. These include:
- your body weight
- your age
- the level of cystine in your white blood cells
- other medical conditions you may have
The following information describes dosages that are commonly used or recommended. However, be sure to take the dosage your doctor prescribes for you. Your doctor will determine the best dosage to fit your needs.
Forms and strengths
Procysbi is available as delayed-release oral capsules and delayed-release oral granules. (“Delayed-release” means the medication is released into your body after it passes through your stomach.) It comes in the following strengths:
- delayed-release oral capsules: 25 milligrams (mg) and 75 mg
- delayed-release oral granules: 75 mg and 300 mg
Recommended dosage
Your doctor will prescribe the dosage of Procysbi that’s right for you. Keep reading to learn more.
Dosage for nephropathic cystinosis
For treatment of nephropathic cystinosis, your doctor will determine your dosage of Procysbi based on your body weight in kilograms (kg) and whether you have taken cysteamine before. (Cysteamine is the active drug in Procysbi.) For reference, one kg is about 2.2 pounds (lb).
If you haven’t taken cysteamine before, your doctor will likely start you on a low dosage of Procysbi and gradually increase this over time to help minimize side effects. The table below describes the typical starting and maintenance dosages of Procysbi.
| Body weight | Starting dosage | Maintenance dosage |
|---|---|---|
| 5 kg or less | 25 or 50 mg every 12 hours | 200 mg every 12 hours |
| 6 to 10 kg | 50 or 75 mg every 12 hours | 300 mg every 12 hours |
| 11 to 15 kg | 75 or 100 mg every 12 hours | 400 mg every 12 hours |
| 16 to 20 kg | 100 or 125 mg every 12 hours | 500 mg every 12 hours |
| 21 to 25 kg | 100 or 150 mg every 12 hours | 600 mg every 12 hours |
| 26 to 30 kg | 125 or 175 mg every 12 hours | 700 mg every 12 hours |
| 31 to 40 kg | 125 or 200 mg every 12 hours | 800 mg every 12 hours |
| 41 to 50 kg | 150 or 225 mg every 12 hours | 900 mg every 12 hours |
| 51 kg or more | 175 or 250 mg every 12 hours | 1000 mg every 12 hours |
If you’re switching to Procysbi from a different form of cysteamine, Cystagon, your doctor will likely prescribe the same total daily dose of Procysbi as what you were taking with Cystagon. But, you’ll split that total daily dose and take it in two doses, 12 hours apart from each other, while taking Procysbi.
To see how well the medication if working for you, your doctor will monitor the amount of cystine in your white blood cells.
Children’s dosage
Procysbi is used in children ages 1 year and older to treat nephropathic cystinosis. The recommended dosages of Procysbi in children is the same as adults. Their dosage will depend on your child’s body weight and whether they’ve taken cysteamine before. See “Dosage for nephropathic cystinosis” above for details.
Talk with your child’s doctor if you have questions about their dosage.
About taking Procysbi
The following are some common questions about taking/using Procysbi.
- Can Procysbi be chewed, crushed, or split? No, you should not chew or crush Procysbi capsules. You should swallow them whole. If you have trouble swallowing capsules, you can open the Procysbi capsule and sprinkle the pellets within onto a small amount of applesauce, berry juice, or fruit juice. Do not mix them in grapefruit juice. Procysbi granules can be taken in the same way by sprinkling them on these foods. Don’t crush or chew the granules.
- Should I take Procysbi with food? In most cases, you should avoid eating food for 2 hours before and 30 minutes after taking Procysbi. In some cases, if you are unable to take Procysbi without food, you can eat up to half a cup of food in the hour before or hour after taking Procysbi. Talk with your doctor or pharmacist for more information about how to take Procysbi with food.
- Is there a best time of day to take Procysbi? Procysbi is taken two times per day, about 12 hours apart. It’s best to take it around the same time each day. This helps keep a steady level of the drug in your body, which helps Procysbi work effectively.
- What if I miss a dose of Procysbi? If you miss a dose of Procysbi and it is more than 4 hours until your next dose, take it as soon as you remember. But if it’s less than 4 hours until your next dose, skip the missed dose and take the next dose at its usual time. You should not take two doses at once to make up for a missed dose. Doing so could raise your risk of side effects.
- Will I need to use Procysbi long term? Procysbi is typically used as a long-term treatment. If you and your doctor determine that it’s safe and effective for your condition, you’ll likely take it long term.
Overdose
Do not takemore Procysbi than your doctor prescribes, as this can lead to harmful effects.
Symptoms caused by an overdose can include:
- nausea or vomiting
- dehydration
What to do in case of overdose
Call your doctor if you think you’ve taken too much Procysbi. You can also call 800-222-1222 to reach America’s Poison Centers or use its online resource. But if you have severe symptoms, immediately call 911 or your local emergency number. Or go to the nearest emergency room.
Procysbi can interact with several other medications and certain foods.
Before taking Procysbi, talk with your doctor and pharmacist. Tell them about all prescription, over-the-counter, and other drugs you take. Also tell them about any vitamins, herbs, or supplements you use. Sharing this information can help you avoid potential interactions.
Interactions with medications and foods
The following table highlights medications and foods that can interact with Procysbi. This list does not contain all drugs that may interact with Procysbi. If you have questions about interactions that may affect you, ask your doctor or pharmacist.
| Medications that can interact with Procysbi | • H2 blockers, such as famotidine (Pepcid AC) • antacids, such as calcium carbonate (Tums) or calcium carbonate/magnesium hydroxide (Rolaids) • proton pump inhibitors (PPIs), such as esomeprazole (Nexium) |
| Foods that can interact with Procysbi | grapefruit |
Procysbi and alcohol
It is not safe to consume alcohol while taking Procysbi. If you have questions about the safety of drinking alcohol while taking Procysbi, talk with your doctor or pharmacist.
Whether you have health insurance or not, cost may be a factor when you’re considering Procysbi. What you’ll pay for Procysbi may depend on several things, such as your treatment plan and the pharmacy you use
Here are a few things to consider regarding cost:
- Comparing pharmacies: The price of Procysbi may vary depending on which pharmacy you use, so it can help to compare prices across pharmacies to find the lowest cost.
- Savings program: A program called Amgen By Your Side may be available. For more information and to find out whether you’re eligible for support, call 855-888-4004 or visit the program website.
If you have questions about how to pay for your prescription, talk with your doctor or pharmacist.
Optum Perks is owned by RVO Health. By clicking on this link, we may receive a commission. Learn more.
The following is important information to consider and discuss with your doctor or pharmacist before taking/using Procysbi.
Pregnancy and Procysbi
It’s not known whether Procysbi is safe to take during pregnancy. If you’re planning a pregnancy or can become pregnant, talk with your doctor before taking/using this drug.
Birth control and Procysbi
It’s not known whether Procysbi is safe to take during pregnancy. Therefore, if you’re sexually active and you or your partner can become pregnant, talk with your doctor about your birth control options while you’re taking/using Procysbi.
Procysbi and breastfeeding
Procysbi should not be taken while breastfeeding. If you’re currently breastfeeding or planning to do so, talk with your doctor about your options.
Before taking Procysbi, discuss your health history with your doctor. Procysbi may not be right for you if you have certain medical conditions or other factors affecting your health. Be sure to talk with your doctor if any of the following apply to you:
- liver problems
- bone problems
- history of bleeding or ulcers in your stomach or intestine
- history of brain or nervous system problems
- low white blood cell count
- skin rash
- previous allergic reaction to this or a similar drug
- pregnancy
- breastfeeding
- alcohol consumption
Note: For more information about the potential negative effects of Procysbi, see the “Side effects of Procysbi” section.
Cysteamine (Cystagon) is another drug available to treat your condition. If you’d like to explore an alternative to Procysbi, talk with your doctor. They can tell you about other medications that might work well for you.
Disclaimer: Healthline has made every effort to make certain that all information is factually correct, comprehensive, and up to date. However, this article should not be used as a substitute for the knowledge and expertise of a licensed healthcare professional. You should always consult your doctor or another healthcare professional before taking any medication. The drug information contained herein is subject to change and is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. The absence of warnings or other information for a given drug does not indicate that the drug or drug combination is safe, effective, or appropriate for all patients or all specific uses.