Cysteamine (Oral)

Cysteamine is used to prevent harm that may occur from the buildup of cystine crystals in organs like the kidneys (nephropathic cystinosis). Cystinosis is a genetic condition that leads to a buildup of the amino acid cystine in cells, forming crystals that can accumulate and damage the kidneys. This medicine helps by removing the excess cystine from the cells in the body.

This medicine is available only with your doctor's prescription.

It is important for your doctor to check your progress at regular visits to make sure this medicine is working properly. Blood tests and eye exams will be needed to look for unwanted effects.

Using high doses of this medicine may cause a condition called Ehler-Danlos-like syndrome. Contact your doctor immediately if you notice unusual changes in your skin and bones, such as bone pain, tenderness, or aching, a curved spine, loss of appetite, stretch marks, broken bones, joint problems, or unusual weight loss.

Serious skin reactions (e.g., erythema multiforme bullosa, toxic epidermal necrolysis) can happen with this medicine. Contact your doctor immediately if you experience blistering, peeling, or loosening of the skin, chills, cough, diarrhea, itching, joint or muscle pain, red irritated eyes, red skin lesions, sore throat, sores, ulcers on the skin, white spots in the mouth or on the lips, or unusual tiredness or weakness while using this medicine.

This medicine may cause stomach or bowel ulcers or bleeding. Contact your doctor immediately if you have severe stomach pain, nausea, vomiting, loss of appetite, or if you are vomiting blood that looks like coffee grounds.

Contact your doctor immediately if you have bloody or persistent diarrhea, loss of bowel control, stomach pain, or vomiting. These may be symptoms of fibrosing colonopathy.

This medicine may affect how your brain functions. Tell your doctor immediately if you experience seizures, depression, or if you become very sleepy while taking this medicine.

Contact your doctor immediately if you notice a bulging soft spot on an infant's head, changes in your ability to see colors, especially blue or yellow, dizziness, headache, hearing loss, persistent ringing in the ears, nausea, blurred or double vision, loss of vision, pain behind the eyes, or pain with eye movement. These may be symptoms of benign intracranial hypertension.

This medicine will reduce the number of some types of blood cells in your body. Because of this, you may get infections more easily. To help with these issues, avoid close contact with people who are sick or have infections. Wash your hands frequently.

This medicine may cause some people to feel dizzy or drowsy. Do not drive or do anything else that could be dangerous until you know how this medicine affects you.

Do not drink alcohol while using this medicine.

It is important to inform your doctor immediately if you are pregnant or become pregnant while using this medicine.

In deciding to use a medicine, you and your doctor need to consider the risks of taking the medicine against the benefits it will provide. For this medicine, keep the following in mind:

Allergies

Inform your doctor if you have ever experienced any unusual or allergic reactions to this medicine or any other medicines. Also, let your healthcare professional know if you have other allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Pediatric

Current studies have not shown any specific problems in children aged 1 year and older that would limit the usefulness of cysteamine. However, safety and effectiveness have not been established for children younger than 1 year.

Geriatric

There is no information available about how age affects the effects of cysteamine in older adults.

Breast Feeding

There are no adequate studies in women to determine the risk to infants when using this medication while breastfeeding. Consider the potential benefits and risks before taking this medication during breastfeeding.

Interactions with Medicines

Some medicines should never be used together, while others can be used together even if an interaction might happen. In these situations, your doctor may adjust the dose or take other precautions. Inform your healthcare professional if you are taking any other prescription or nonprescription (over-the-counter [OTC]) medicines.

Interactions with Food/Tobacco/Alcohol

Certain medicines should not be used around the time of eating food or specific types of food because interactions may occur. Using alcohol or tobacco with certain medicines can also cause interactions. The following interactions are noted for their potential significance but may not be a complete list.

Using this medicine with any of the following is generally not recommended, but may be necessary in some cases. If used together, your doctor may adjust the dose or frequency of this medicine or provide special instructions regarding the use of food, alcohol, or tobacco.

• Ethanol

Other Medical Problems

Having other medical problems may impact the use of this medicine. Be sure to inform your doctor about any other medical issues, especially:

• Blood problems, or history of or
• Bone problems or
• Brain disease or
• Depression, history of or
• Head injury, history of or
• Increased pressure in the head or
• Liver disease or
• Seizures, history of or
• Stomach or bowel problems (e.g., ulcers, bleeding)—Use with caution. This may worsen these conditions.

Along with its intended benefits, a medicine may cause some unwanted effects. Not all of these side effects may happen, but if they do, you may need medical attention.

Check with your doctor immediately if any of the following side effects happen:

More common

• Diarrhea
• drowsiness
• fever
• loss of appetite
• nausea
• skin rash
• stomach pain
• vomiting

Less common

• Confusion
• dizziness
• headache
• mental depression
• sore throat
• trembling

Rare

• Increased thirst
• seizures
• unusual tiredness or weakness

Incidence not known

• Blistering, peeling, or loosening of the skin
• blurred or double vision
• bone lesions
• bulging soft spot on the head of an infant
• change in the ability to see colors, especially blue or yellow
• chills
• continuing ringing or buzzing or other unexplained noise in the ears
• cough
• eye pain
• hearing loss
• itching
• joint or muscle pain
• loss of appetite
• loss of bowel control
• loss of vision
• pain behind the eyes
• pain in the legs
• pain with eye movement
• red skin lesions, often with a purple center
• red, irritated eyes
• reddish purple lines on the arms, face, legs, trunk, or groin
• severe headache
• sores, ulcers, or white spots in the mouth or on the lips

Some side effects may happen that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue, are bothersome, or if you have any questions about them:

Less common

• Breath odor
• constipation
• hives
• redness of the skin
• unusual drowsiness, dullness, tiredness, weakness, or feeling of sluggishness
• weight loss

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

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

In the U.S.

• Cystagon
• Procysbi

Available Dosage Forms:

• Capsule, Delayed Release
• Capsule
• Packet

Therapeutic Class: Renal-Urologic Agent

Take this medicine only as directed by your doctor. Do not take more of it, do not take it more often, and do not take it for a longer time than your doctor ordered. To do so may increase the chance of side effects.

This medicine comes with a patient information leaflet. Read and follow the instructions carefully. Ask your doctor or pharmacist if you have any questions.

If you vomit your dose of cysteamine within 20 minutes of taking it, take the dose again. However, if you vomit the dose a second time, do not repeat the dose but wait and take your next dose as scheduled. Also, if vomiting occurs more than 20 minutes after you take your dose, do not repeat the dose.

For children 1 year of age and older, the capsule may be opened and the contents of the capsule sprinkled on food or mixed in formula.

Swallow the delayed-release capsule or granules whole. Do not crush or chew the capsule or contents of the capsule or packet.

Take the delayed-release capsule or granules the same way every day. This means take it at the same time and take it consistently, either with or without food. Avoid high-fat meals close to the time that you will take this medicine.

• Without food: Take it at least 30 minutes before meals or at least 2 hours after meals.
• With food: You can eat a small amount of food (½ cup) between 1 hour before and after taking Procysbi®.

If you cannot swallow the capsule whole, you may open it and take the capsule or packet contents with certain foods and juices or water:

• Taking this medicine with applesauce or berry jelly: Open the capsule or packet and sprinkle the granules on 4 ounces (½ cup) of applesauce or berry jelly. Mix the granules into the food. Swallow the entire mixture within 30 minutes. Do not chew the granules. Do not save the applesauce or berry jelly and granules for later use.
• Taking this medicine with fruit juice (except grapefruit juice): Open the capsule or packet and sprinkle the granules into 4 ounces (½ cup) of juice. Gently stir until mixed. Drink it within 30 minutes. Do not chew the granules. Do not save the fruit juice or granules for later use.
• If you are using a gastrostomy tube: Open the capsule or packet and sprinkle the granules on 4 ounces (½ cup) of applesauce. For children receiving 1 or 2 capsules, use a minimum of 1 ounce of applesauce. Use only strained applesauce with no chunks. Mix and give the medicine through the gastrostomy tube into the stomach within 30 minutes. Flush the tube with 10 milliliters (mL) of fruit juice or water. Do not save the mixture for later use.

It is important that you follow any special instructions from your doctor, such as taking dietary supplements, vitamin D, and thyroid hormone. These supplements will replace minerals lost through the kidneys.

If you are taking medicines containing bicarbonate or carbonate, take Procysbi® at least 1 hour before or after these medicines.

Dosing

The dose of this medicine will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so.

The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.

• To prevent buildup of cystine crystals in the kidneys: For oral dosage form (capsules): Adults—The starting dose must be determined by your doctor. Your doctor may gradually increase your dose. Children—The starting dose is based on body size and must be determined by your doctor. Your doctor may gradually increase your dose. For oral dosage form (delayed-release capsules or granules): For patients not on cysteamine therapy: Adults—The starting dose is based on body size and must be determined by your doctor. The capsules are taken every 12 hours. Your doctor may adjust your dose as needed. Children 1 year of age and older—The starting dose is based on body size and must be determined by your doctor. The capsules are taken every 12 hours. Your doctor may adjust your dose as needed. Children younger than 1 year of age—Use and dose must be determined by your doctor. For patients switching from cysteamine immediate-release capsules: Adults—The total dose is the same as the total dose that you are currently taking. Divide the total daily dose by 2 and take it every 12 hours. Children 1 year of age and older—The total dose is the same as the total dose that you are currently taking. Children younger than 1 year of age—Use and dose must be determined by your doctor.

Missed Dose

If you miss a dose of this medicine, take it as soon as possible. However, if it is almost time for your next dose, skip the missed dose and go back to your regular dosing schedule. Do not double doses.

You may take a missed dose of Procysbi® as soon as you can up to 8 hours after your scheduled dose. If you missed a dose of Procysbi® and your next dose is less than 4 hours away, wait and take your next dose at the normal time. Do not take 2 doses at one time to make up for a missed dose.

Storage

Keep out of the reach of children.

Do not keep outdated medicine or medicine no longer needed.

Ask your healthcare professional how you should dispose of any medicine you do not use.

Store the medicine in a closed container at room temperature, away from heat, moisture, and direct light. Keep from freezing.

Store the delayed-release capsule in its original container. Keep it in a dry place away from direct light.