"The U.S. Food and Drug Administration today approved Procysbi (cysteamine bitartrate) for the management of nephropathic cystinosis in children and adults. Procysbi was granted orphan product designation because it is intended to treat a rare dis"...
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Details with Side Effects
In three clinical trials, cysteamine or phosphocysteamine have been administered to 246 children with cystinosis. Causality of side effects is sometimes difficult to determine because adverse effects may result from the underlying disease.
The most frequent adverse reactions seen involve the gastrointestinal and central nervous systems. These are especially prominent at the initiation of cysteamine therapy. Temporarily suspending treatment, then gradual re-introduction may be effective in improving tolerance.
Adverse reactions were not collected systematically in the NCCS, but were often listed by investigators. The following rates may therefore be underestimated. The most common events (> 5%) were vomiting 35%, anorexia 31%, fever 22%, diarrhea 16%, lethargy 11%, and rash 7%.
Less common adverse events are:
Body as a whole: Dehydration.
Psychiatric: Nervousness, abnormal thinking, depression, emotional lability, hallucinations, nightmares.
Adverse reactions or intolerance leading to cessation of treatment occurred in 8% of patients in the U.S. Studies.
Withdrawals due to intolerance, vomiting associated with medication, anorexia, lethargy, and fever appeared dose related, occurring more frequently in those patients receiving 1.95 grams/m2/day as compared to 1.30 grams/m2/day.
|Dose in Grams/m2/day|
(n = 42)
(n = 51)
|Vomiting Considered Related to Medicine||31||67|
Sudden deaths have been reported in this disease state.
Benign intracranial hypertension (or pseudotumor cerebri; PTC) with papilledema; skin lesions, molluscoid pseudotumors, skin striae, skin fragility; joint hyperextension, leg pain, genu valgum, osteopenia, compression fracture and scoliosis have been reported (see PRECAUTIONS).
Drug Abuse and Dependence
CYSTAGON® (cysteamine bitartrate) has not been associated with abuse potential, psychological or physical dependence in humans.
Read the Cystagon (cysteamine bitartrate) Side Effects Center for a complete guide to possible side effects
None have been described. CYSTAGON® (cysteamine bitartrate) can be administered with electrolyte and mineral replacements necessary for management of the Fanconi Syndrome as well as vitamin D and thyroid hormone.
Read the Cystagon Drug Interactions Center for a complete guide to possible interactions
Last reviewed on RxList: 6/26/2007
This monograph has been modified to include the generic and brand name in many instances.
Additional Cystagon Information
Report Problems to the Food and Drug Administration
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit the FDA MedWatch website or call 1-800-FDA-1088.
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