IMPORTANT SAFETY INFORMATION
What is PROCYSBI (Pro-CIS-bee)?
PROCYSBI is a prescription medicine used to manage a medical condition called nephropathic cystinosis in adults and children 6 years of age and older.
It is not known if PROCYSBI is safe and effective in children under 6 years of age.
Who should not take PROCYSBI?
Do not take PROCYSBI if you are allergic to penicillamine.
What should I tell my doctor before taking PROCYSBI?
Before you take PROCYSBI, tell your doctor if you:
- have a skin rash or bone problems
- have a history of seizures, exhaustion, sleepiness, depression, or other nervous system problems
- have or have had stomach or bowel (intestinal) problems including ulcers or bleeding
- 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.
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.
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. 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.
- 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.
- 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.
- 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:
- abdominal pain or discomfort
- loss of appetite or decreased appetite
- breath odor
- skin odor
- skin rash
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. 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.
To report a PROCYSBI Product Adverse Event or Product Complaint contact Raptor Pharmaceuticals Inc.:
Email firstname.lastname@example.org or call 1-855-888-4004, option 2
Please see Full Prescribing Information and Patient Package Insert.
INDICATIONS AND USAGE: PROCYSBI® (cysteamine bitartrate) delayed-release capsules is a cystine depleting agent indicated for the management of nephropathic cystinosis in adults and children ages 6 years and older.
- Hypersensitivity to penicillamine.
IMPORTANT SAFETY INFORMATION
WARNING AND PRECAUTIONS:
- Ehlers-Danlos like Syndrome: Skin and bone lesions that resemble clinical findings for Ehlers-Danlos syndrome have been reported in patients treated with high doses of immediate-release cysteamine bitartrate or other cysteamine salts.
- These include molluscoid pseudotumors (purplish hemorrhagic lesions), skin striae, bone lesions (including osteopenia, compression fractures, scoliosis and genu valgum), leg pain, and joint hyperextension.
- One patient on immediate-release cysteamine bitartrate with serious skin lesions subsequently died of acute cerebral ischemia with marked vasculopathy.
- Monitor patients for development of skin or bone lesions and interrupt PROCYSBI dosing if patients develop these lesions. PROCYSBI may be restarted at a lower dose under close supervision, then slowly increased to the appropriate therapeutic dose.
- Skin Rash: Severe skin rashes such as erythema multiforme bullosa or toxic epidermal necrolysis have been reported in patients receiving immediate-release cysteamine bitartrate. If severe skin rashes develop, discontinue use of any cysteamine product permanently.
- Gastrointestinal Ulcers and Bleeding: Gastrointestinal (GI) ulceration and bleeding have been reported in patients receiving immediate-release cysteamine bitartrate.
- GI tract symptoms including nausea, vomiting, anorexia and abdominal pain, sometimes severe, have been associated with cysteamine. If severe GI tract symptoms develop, consider decreasing the dose of PROCYSBI.
- Central Nervous System Symptoms: Central Nervous System (CNS) symptoms such as seizures, lethargy, somnolence, depression, and encephalopathy have been associated with immediate-release cysteamine.
- Neurological complications have also been described in some patients with cystinosis who have not been treated with cysteamine.
- Carefully evaluate and monitor patients who develop CNS symptoms. Interrupt or adjust the dose as necessary for patients with severe symptoms or with symptoms that persist or progress.
- Patients should exercise caution when driving or engaging in other hazardous activities when taking cysteamine.
- Leukopenia and Elevated Alkaline Phosphatase Levels: Cysteamine has been associated with reversible leukopenia and elevated alkaline phosphatase levels. Therefore, blood counts and alkaline phosphatase levels should be monitored.
- Benign Intracranial Hypertension: Benign intracranial hypertension (pseudotumor cerebri; PTC) and/or papilledema has been reported in patients receiving immediate-release cysteamine bitartrate treatment.
- A causal relationship between PTC and cysteamine has not been established.
- Physicians should monitor patients for signs and symptoms of PTC, including headache, tinnitus, dizziness, nausea, diplopia, blurry vision, loss of vision, pain behind the eye or pain with eye movement.
The most common reactions (≥5%):
- vomiting (35%)
- anorexia (31%)
- fever (22%)
- diarrhea (16%)
- lethargy (11%)
- rash (7%)
Other adverse reactions included:
- bad breath
- abdominal pain
To report SUSPECTED ADVERSE REACTIONS, contact Raptor Pharmaceuticals Inc. at 1-855-888-4004 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
- PROCYSBI can be administered with electrolyte (except bicarbonate) and mineral replacements necessary for management of Fanconi Syndrome as well as vitamin D and thyroid hormone.
USE IN SPECIFIC POPULATIONS
- Breastfeeding is not recommended while taking PROCYSBI.
Please see Full Prescribing Information.