CYSTEAMINE THERAPY WITH 12-HOUR DOSING

What is PROCYSBI?

PROCYSBI® (cysteamine bitartrate) delayed-release capsules are the first and only cysteamine therapy with 12-hour dosing approved for the management of nephropathic cystinosis in adults and children at least one year old.

PROCYSBI helps reduce cystine levels with 2 doses in 24 hours, taken every 12 hours at the same time every day, allowing patients and caregivers to choose consistent times that work for them. For example, you might take PROCYSBI at 7:00 a.m. and again at 7:00 p.m. every day. This means you can make a routine that fits your schedule.

Why Patient Why Patient Why Patient

Clinical trials with PROCYSBI

In clinical trials with people with nephropathic cystinosis who took PROCYSBI every 12 hours as directed:

  • PROCYSBI controlled cystine levels for a full 12 hours
  • PROCYSBI maintained kidney function during 24 months or more of treatment
  • The safety and effectiveness of PROCYSBI have been established in children 1 year of age and older; cystine levels were maintained through the 18 months of the trial
  • PROCYSBI improved weight and height in children older than 1 year and younger than 6 years (comparisons were made based on the change in weight and height from day 1 to 12 months and 18 months of the trial)

How PROCYSBI works

How PROCYSBI works graphic

PROCYSBI is a delayed-release formulation of cysteamine bitartrate that was designed to release in the small intestine instead of the stomach. A coating on the microbeads helps control how and where the medicine is released in the body. The coating does not break down in acidic environments, such as the stomach. Instead it breaks down in less acidic environments like the small intestine.

PROCYSBI passes through the stomach before it reaches the small intestine. Once in the small intestine, the medicine in PROCYSBI is gradually released over 12 hours–just long enough for the next dose of PROCYSBI to reach the small intestine. This gradual release of cysteamine ensures continuous dosing.

When acid levels in your stomach are higher, the microbead coating works as it should and doesn’t release the medicine until it reaches the small intestine. What you eat and drink can affect acid levels. If acid levels are too low, it can result in the medicine releasing too soon (in the stomach).

Do not drink alcohol if you take PROCYSBI. Drinking alcohol while taking PROCYSBI may change how PROCYSBI works and may cause an increase in the amount of PROCYSBI in your blood that may cause serious side effects.

When taking PROCYSBI as directed, continuous cystine control may be achieved. Talk to your doctor about how to take PROCYSBI with foods that have an appropriate level of acidity to ensure that it doesn’t dissolve in the stomach.

APPROVED USES and IMPORTANT SAFETY INFORMATION for PROCYSBI

What is PROCYSBI?

PROCYSBI (cysteamine bitartrate) delayed-release capsules is a prescription medicine used to treat a medical condition called nephropathic cystinosis in adults and children 1 year of age and older. It is not known if PROCYSBI is safe and effective in children under 1 year of age.

IMPORTANT SAFETY INFORMATION

What is the most important safety information I should know about PROCYSBI?

PROCYSBI can cause serious side effects, including:

  • Skin, bone, and joint problems. People treated with high doses of cysteamine bitartrate may develop abnormal changes of their skin and bones, such as stretch marks, bone injuries (such as fractures), bone deformities, and joint problems. Check your skin while taking PROCYSBI. Tell your doctor if you notice any skin changes or problems with your bones or joints. Your doctor will check you for these problems.
  • Skin rash. Skin rash is common with cysteamine bitartrate and may sometimes be severe. Your dose of PROCYSBI may need to be decreased until the rash goes away. If the rash is severe, your doctor may tell you to stop taking PROCYSBI.  Tell your doctor right away if you get a skin rash.
  • Stomach and bowel (intestinal) problems. Some people who take other medicines that contain cysteamine bitartrate develop ulcers and bleeding in their stomach or bowel. Tell your doctor right away if you get stomach-area pain, nausea, vomiting, loss of appetite, or vomit blood.
  • Central nervous system symptoms. Some people who take other medicines that contain cysteamine bitartrate develop seizures, depression, and become very sleepy. The medicine may affect how your brain is working (encephalopathy). Tell your doctor right away if you develop any of these symptoms.
  • Low white blood cell count and certain abnormal liver function blood tests. Your doctor should check you for these problems.
  • Benign intracranial hypertension (pseudotumor cerebri) has happened in some people who take immediate-release cysteamine bitartrate. This is a condition where there is high pressure in the fluid around the brain. Your doctor should do eye examinations to find and treat this problem early. Tell your doctor right away if you develop any of the following symptoms while taking PROCYSBI: headache, buzzing or "whooshing" sound in the ear, dizziness, nausea, double vision, blurry vision, loss of vision, pain behind the eye, or pain with eye movement.

Who should not take PROCYSBI?

Do not take PROCYSBI if you are allergic to penicillamine or cysteamine.

What should I tell my doctor before taking PROCYSBI?

Tell your doctor if you have any other medical conditions, including if you:

  • drink alcohol.
  • have a skin rash or bone problems.
  • have or have had stomach or bowel (intestinal) problems including ulcers or bleeding.
  • have a history of seizures, lack of energy, unusual sleepiness, depression, or changes in your ability to think clearly.
  • have liver or blood problems.
  • are pregnant or plan to become pregnant. It is not known if PROCYSBI will harm your unborn baby. Tell your doctor right away if you think that you are pregnant. Talk with your doctor about the benefits and risks of taking PROCYSBI during pregnancy.
  • are breastfeeding or plan to breastfeed. You should not breastfeed during treatment with PROCYSBI. Talk with your doctor about the best way to feed your baby if you take PROCYSBI.

What should I avoid while taking PROCYSBI?

  • Do not drive or operate machinery until you know how PROCYSBI affects you. PROCYSBI can make you sleepy or less alert than normal.
  • Do not drink alcohol if you take PROCYSBI. Drinking alcohol while taking PROCYSBI may change how PROCYSBI works and may cause an increase in the amount of PROCYSBI in your blood that may cause serious side effects.

What are the possible side effects of PROCYSBI?

  • See “What is the most important information I should know about PROCYSBI?”

The most common side effects of PROCYSBI include: vomiting, nausea, stomach (abdominal) pain, breath odor, diarrhea, skin odor, tiredness, skin rash, headache, problems with body salts or electrolytes.

These are not all of the possible side effects of PROCYSBI. Call your doctor for medical information about side effects.

For additional important safety information, click here for the Full Prescribing InformationPatient Information, and Instructions for Use and discuss with your doctor.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit http://www.fda.gov/medwatch or call 1-800-FDA-1088.

APPROVED USES and IMPORTANT SAFETY INFORMATION for PROCYSBI

What is PROCYSBI?

PROCYSBI (cysteamine bitartrate) delayed-release capsules is a prescription medicine used to treat a medical condition called nephropathic cystinosis in adults and children 1 year of age and older. It is not known if PROCYSBI is safe and effective in children under 1 year of age.

IMPORTANT SAFETY INFORMATION

What is the most important safety information I should know about PROCYSBI?

PROCYSBI can cause serious side effects, including:

  • Skin, bone, and joint problems. People treated with high doses of cysteamine bitartrate may develop abnormal changes of their skin and bones, such as stretch marks, bone injuries (such as fractures), bone deformities, and joint problems. Check your skin while taking PROCYSBI. Tell your doctor if you notice any skin changes or problems with your bones or joints. Your doctor will check you for these problems.
  • Skin rash. Skin rash is common with cysteamine bitartrate and may sometimes be severe. Your dose of PROCYSBI may need to be decreased until the rash goes away. If the rash is severe, your doctor may tell you to stop taking PROCYSBI.  Tell your doctor right away if you get a skin rash.
  • Stomach and bowel (intestinal) problems. Some people who take other medicines that contain cysteamine bitartrate develop ulcers and bleeding in their stomach or bowel. Tell your doctor right away if you get stomach-area pain, nausea, vomiting, loss of appetite, or vomit blood.
  • Central nervous system symptoms. Some people who take other medicines that contain cysteamine bitartrate develop seizures, depression, and become very sleepy. The medicine may affect how your brain is working (encephalopathy). Tell your doctor right away if you develop any of these symptoms.
  • Low white blood cell count and certain abnormal liver function blood tests. Your doctor should check you for these problems.
  • Benign intracranial hypertension (pseudotumor cerebri) has happened in some people who take immediate-release cysteamine bitartrate. This is a condition where there is high pressure in the fluid around the brain. Your doctor should do eye examinations to find and treat this problem early. Tell your doctor right away if you develop any of the following symptoms while taking PROCYSBI: headache, buzzing or "whooshing" sound in the ear, dizziness, nausea, double vision, blurry vision, loss of vision, pain behind the eye, or pain with eye movement.

Who should not take PROCYSBI?

Do not take PROCYSBI if you are allergic to penicillamine or cysteamine.

What should I tell my doctor before taking PROCYSBI?

Tell your doctor if you have any other medical conditions, including if you:

  • drink alcohol.
  • have a skin rash or bone problems.
  • have or have had stomach or bowel (intestinal) problems including ulcers or bleeding.
  • have a history of seizures, lack of energy, unusual sleepiness, depression, or changes in your ability to think clearly.
  • have liver or blood problems.
  • are pregnant or plan to become pregnant. It is not known if PROCYSBI will harm your unborn baby. Tell your doctor right away if you think that you are pregnant. Talk with your doctor about the benefits and risks of taking PROCYSBI during pregnancy.
  • are breastfeeding or plan to breastfeed. You should not breastfeed during treatment with PROCYSBI. Talk with your doctor about the best way to feed your baby if you take PROCYSBI.

What should I avoid while taking PROCYSBI?

  • Do not drive or operate machinery until you know how PROCYSBI affects you. PROCYSBI can make you sleepy or less alert than normal.
  • Do not drink alcohol if you take PROCYSBI. Drinking alcohol while taking PROCYSBI may change how PROCYSBI works and may cause an increase in the amount of PROCYSBI in your blood that may cause serious side effects.

What are the possible side effects of PROCYSBI?

  • See “What is the most important information I should know about PROCYSBI?”

The most common side effects of PROCYSBI include: vomiting, nausea, stomach (abdominal) pain, breath odor, diarrhea, skin odor, tiredness, skin rash, headache, problems with body salts or electrolytes.

These are not all of the possible side effects of PROCYSBI. Call your doctor for medical information about side effects.

For additional important safety information, click here for the Full Prescribing InformationPatient Information, and Instructions for Use and discuss with your doctor.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit http://www.fda.gov/medwatch or call 1-800-FDA-1088.