April 30, 2017
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Side Effects


The following adverse reactions are also discussed in other sections of the labeling:

Clinical Trials Experience

Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared with rates in the clinical trials of another drug and may not reflect the rates observed in clinical practice.

The data described below reflect exposure to cysteamine in 328 patients with nephropathic cystinosis (246 patients receiving immediate-release cysteamine as cysteamine hydrochloride or phosphocysteamine, and 63 patients receiving PROCYSBI) in open-label clinical trials.

Clinical Trials Experience With PROCYSBI

Sixty-two patients with cystinosis (38 males and 24 females) received PROCYSBI in two clinical trials at doses ranging from 0.29 grams/m2 per day to 2.19 grams/m2 per day [see Clinical Studies]. All patients were switched from immediate-release cysteamine to PROCYSBI. Forty-three patients, ages 7 to 24 years old, received PROCYSBI in an 8-week, open-label, randomized, cross-over trial comparing PROCYSBI to immediate-release cysteamine bitartrate. Forty of 43 patients continued PROCYSBI treatment in an open-label extension trial, and were treated with PROCYSBI for longer than 2 years. An additional 19 patients (6 transplanted patients and 13 patients aged 2 to 6 years) were enrolled directly into this trial and were treated with PROCYSBI for up to 18 months.

In the open-label, randomized, cross-over trial, a higher incidence of adverse reactions was reported in patients during the PROCYSBI treatment period compared with the immediate-release cysteamine treatment period (see Table 3). Other significant adverse reactions reported during clinical trials included hypersensitivity reactions, including anaphylaxis.

Table 3: Adverse reactions that occurred in ≥5% of patients in the randomized, cross -over clinical trial

Adverse Reaction Immediate-release cysteamine PROCYSBI
(n = 41)
(n = 43)
Vomiting/emesis 12 19
Nausea 7 16
Abdominal pain/discomfort 0 14
Headache 0 9
Dizziness 0 5
Anorexia/loss of appetite 5 2

For all patients treated with PROCYSBI in both trials (N=62), the most commonly reported adverse reactions (>5%) were vomiting, nausea, abdominal pain, breath odor, diarrhea, skin odor, fatigue, rash, and headache.

Clinical Trials Experience With Immediate-Release Cysteamine

The most frequent adverse reactions involved the gastrointestinal and central nervous systems and were especially prominent at the initiation of cysteamine therapy. Most patients were able to resume therapy at lower doses. The most common reactions (>5%) were vomiting, anorexia, fever, diarrhea, lethargy, and rash. Other adverse reactions included nausea, bad breath, abdominal pain, headache, dizziness, and urticaria.

Withdrawals due to intolerance, vomiting, anorexia, lethargy, and fever occurred more frequently in those patients receiving 1.95 grams/m2 per day as compared with 1.3 grams/m2 per day of immediaterelease cysteamine bitartrate.

Postmarketing Experience

The following adverse reactions have been identified during post-approval use of immediate-release cysteamine bitartrate. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure.

Read the Procysbi (cysteamine bitartrate delayed-release capsules) Side Effects Center for a complete guide to possible side effects


Drugs That Increase Gastric pH

Drugs that increase the gastric pH (e.g., proton pump inhibitors, medications containing bicarbonate or carbonate) may alter the pharmacokinetics of cysteamine due to the premature release of cysteamine from PROCYSBI and increase WBC cystine concentration. Concomitant administration of 20 mg omeprazole did not significantly affect the pharmacokinetics of cysteamine when PROCYSBI was administered with 240 mL of orange juice [CLINICAL PHARMACOLOGY]. The effect of omeprazole on the pharmacokinetics of cysteamine was not studied after PROCYSBI administration with water. Monitor WBC cystine concentration when drugs that increase the gastric pH are concomitantly used [see DOSAGE AND ADMINISTRATION].

Use With Alcohol

Consumption of alcohol with PROCYSBI may increase the rate of cysteamine release and/or adversely alter the pharmacokinetic properties, as well as the effectiveness and safety of PROCYSBI. Therefore, do not consume alcoholic beverages during treatment with PROCYSBI [see DOSAGE AND ADMINISTRATION].

Other Medications Used For The Management Of Fanconi Syndrome

PROCYSBI can be administered with other electrolyte and mineral replacements necessary for management of Fanconi syndrome, as well as vitamin D and thyroid hormone.

This monograph has been modified to include the generic and brand name in many instances.

Last reviewed on RxList: 12/28/2016

Side Effects

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