What Is PROCYSBI?

PROCYSBI® (cysteamine bitartrate) delayed-release capsules is the first FDA-approved, cystine-depleting medication dosed every 12 hours

PROCYSBI is a delayed-release form of cysteamine bitartrate that works by continuously reducing the toxic concentration of cystine in the cells.1 Keeping cystine concentration under control is the only way to limit or prevent the damage too much cystine can cause to cells, tissue, and organs.2-6

PROCYSBI plays an important role in the management of nephropathic cystinosis. PROCYSBI provides continuous cystine control when taken every 12 hours. That means patients and caregivers only have to think about dosing twice a day—so they can design a routine that fits their unique lifestyle.1,7

PROCYSBI now offers multiple administration options

Download the full Prescribing Information to learn more

Why PROCYSBI?

Continuous cystine control with twice-daily dosing, given every 12 hours

Flexibility

Taking cystine-depleting medicine regularly and on a strict schedule is a critical part of successfully managing cystinosis because even brief interruptions in a treatment routine can result in a dangerous buildup of cystine.2,4,5

PROCYSBI works to control cystine concentration continuously over 12 hours. That means patients and caregivers only have to think about dosing twice a day—so they can choose a schedule that fits their lifestyle.1,7 For example, if a patient takes a first dose of PROCYSBI at 7 a.m., he or she would take the second dose at exactly 7 p.m. Twelve-hour control may also eliminate the need to wake up at night to take a dose.

Combined with consistent white blood cell cystine testing, PROCYSBI can help you confidently maintain your cystine concentration.1,7

There are also several different ways that you can take your PROCYSBI medication. It can be swallowed, mixed with approved food or beverages, or administered through a G-tube (feeding tube). This gives you even more flexibility around how you control your cystine concentration.1 Talk to your doctor about a dosing schedule that best fits your life.

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In a research study, people with cystinosis who took PROCYSBI every 12 hours as directed maintained white blood cell concentrations and showed no worsening of kidney function during 24 months or more of treatment.1,7,8

PROCYSBI uses tiny microbeads that are coated so they bypass the stomach and are more easily absorbed in the small intestine. This means that each dose of PROCYSBI delivers cysteamine slowly and helps control cystine concentration for a full 12 hours.1,7,8

Is PROCYSBI right for me?

If you or your child has cystinosis, answer these simple questions to find out if PROCYSBI can help achieve cystine control

Frequently asked questions about PROCYSBI

  • PROCYSBI is a delayed-release form of cysteamine bitartrate that works by continuously reducing the toxic concentration of cystine in the cells.1 Keeping cystine concentration under control is the only way to limit or prevent the damage too much cystine can cause to cells, tissue, and organs.2-6

    By providing continuous control of cystine when taken every 12 hours, PROCYSBI plays an important role in the management of nephropathic cystinosis.1,7

  • Cystinosis is a progressive, incurable disease. Maintaining healthy cystine concentration (less than 1 nmol ½ cystine/mg of protein) is the only way to prevent severe and irreversible organ damage. This is true even if you’ve had a kidney transplant and even if you look or feel fine.2-6

    Even brief interruptions in your PROCYSBI treatment can allow cystine concentration to become toxic again.5

  • White blood cell (WBC) cystine testing helps you see any changes in your cystine concentrations, which helps you and your doctor determine if you are titrated to maximum clinical benefit.9

    In a research study, people with cystinosis who took PROCYSBI every 12 hours as directed maintained their WBC concentration and kidney function during 24 months or more of treatment.1 Taking PROCYSBI as directed and having regular WBC cystine tests can help you confidently maintain your cystine concentrations.1,7,8

  • PROCYSBI is a cystine-depleting medication that works to continuously lower the amount of cystine in your cells. PROCYSBI uses tiny microbeads that are coated so they bypass the stomach and are more easily absorbed in the small intestine. This means that each dose of PROCYSBI delivers cysteamine slowly and helps control cystine concentration for a full 12 hours.1,7,8

  • Yes. PROCYSBI was approved in the United States in 2013.

    PROCYSBI received FDA approval on an expanded label for the treatment of children aged 2-6 years with nephropathic cystinosis in August, 2015.

     

  • PROCYSBI can be taken in several different ways. You can swallow a PROCYSBI capsule whole, mix it with certain food or drinks, or take it via G-tube. Visit How to take PROCYSBI and talk to your doctor about the best way for you to take it.1

  • In clinical trials, some people (greater than 5%) experienced side effects while taking PROCYSBI. The most common side effects with PROCYSBI include:1

    • Vomiting
    • Nausea
    • Abdominal pain
    • Breath odor
    • Diarrhea
    • Skin odor
    • Fatigue
    • Rash
    • Headache

    The most common side effects involve the gastrointestinal system (digestion) and the central nervous system. This may be especially true at the start of therapy. Your doctor can help you manage these side effects by lowering your dose and gradually increasing it.1

    These are not the only side effects of PROCYSBI. Read more about other possible side effects. Learn more ›

  • US_BOTTLES_TOGETHER
    PROCYSBI is supplied in two different bottles:

    • 25mg capsules, 60 capsules per bottle
    • 75mg capsules, 250 capsules per bottle

    Multiple capsules are needed for each dose. Your doctor will decide what dose is right for you and how many of which bottles you should get.1

  • Only your doctor can prescribe PROCYSBI.

    PROCYSBI is supplied exclusively by a specialty pharmacy, Accredo Specialty Pharmacy.

  • After your doctor prescribes PROCYSBI, you can enroll in TranscendCare™, a patient-support program created with input from people with cystinosis and their caregivers, who understand the challenges you may experience.

    For eligible patients TranscendCare offers assistance to help you with insurance coverage and financial support, answer questions you have about PROCYSBI, help you order refills, and more.TranscendCare also provides a Patient Access Manager who can answer additional questions about PROCYSBI and offer reimbursement and financial support, including for costs related to diagnostic tests and traveling for treatment. Learn more ›

References

1. PROCYSBI [package insert]. Horizon Orphan LLC; 2015.

2. Brodin-Sartorius A, Tète M-J, Niaudet P, et al. Cysteamine therapy delays the progression of nephropathic cystinosis in late adolescents and adults. Kidney Int. 2012;81:179-189.

3. Gahl WA, Thoene JG, Schneider JA. Cystinosis. N Engl J Med. 2002;347(2):111-121.

4. Gahl WA, Balog JZ, Kleta R. Nephropathic cystinosis in adults: Natural history and effects of oral cysteamine therapy. Ann Intern Med. 2007;147:242-250.

5. Levtchenko EN, van Dael CM, de Graaf-Hess AC, et al. Strict cysteamine dose regimen is required to prevent nocturnal cystine accumulation in cystinosis. Pediatr Nephrol. 2006;21:110-113.

6. Nesterova G, Gahl WA. Cystinosis: The evolution of a treatable disease. Pediatr Nephrol. 2013;28:51-59.

7. Langman CB, Greenbaum LA, Sarwal M, et al. A randomized controlled crossover trial with delayed-release cysteamine bitartrate in nephropathic cystinosis: Effectiveness on white blood cell cystine levels and comparison of safety. Clin J Am Soc Nephrol. 2012;7:1112-1120.

8. Langman CB, Greenbaum LA, Grimm P, et al. Quality of life is improved and kidney function preserved in patients with nephropathic cystinosis treated for 2 years with delayed-release cysteamine bitartrate. J Pediatr. 2014;165(3):528-533.e.1.

9. Dalton, Neil. The importance of accurate cystine level testing. Cystinosis Research Network. Available at https://cystinosis.org/images/research/article-library/cystagon/2009_01_Cystine_Level_Testing.pdf. Accessed December 4, 2015.

 

What is PROCYSBI (Pro-CIS-bee)?

PROCYSBI® (cysteamine bitartrate) delayed-release capsules is a prescription medicine used to treat a medical condition called nephropathic cystinosis, in adults and children 2 years of age and older.

It is not known if PROCYSBI is safe and effective in children under 2 years of age.

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?

Before you take PROCYSBI, tell your doctor 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
  • have any other medical conditions
  • 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.

Tell your doctor about all medicines that you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

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

What should I avoid while taking PROCYSBI?

Do not drive or operate heavy 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? 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. These changes may include 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. Tell your doctor right away if you get a skin rash. 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.
  • 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.

The most common side effects with PROCYSBI include:

  • vomiting
  • nausea
  • stomach (abdominal) pain
  • breath odor
  • diarrhea
  • skin odor
  • tiredness
  • skin rash
  • headache

Tell your doctor 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 PROCYSBI. Call your doctor for medical advice about side effects.

You may report side effects to the FDA at 1-800-FDA-1088.

Issued: 8/2015 Piece number: P-PYB-00033 Derived from Patient Information approved 8/2015