Procysbi

1

ANNEX 1

SUMMARY OF PRODUCT CHARACTERISTICS



2

1. NAME OF THE MEDICINAL PRODUCT

PROCYSBI 25 mg gastro-resistant hard capsules
PROCYSBI 75 mg gastro-resistant hard capsules

2. QUALITATIVE AND QUANTITATIVE COMPOSITION

PROCYSBI 25 mg hard capsule
Each hard capsule contains 25 mg of cysteamine (as mercaptamine bitartrate).

PROCYSBI 75 mg hard capsule
Each hard capsule contains 75 mg of cysteamine (as mercaptamine bitartrate).

For the full list of excipients, see section 6.1.

3. PHARMACEUTICAL FORM

Gastro-resistant hard capsule.

PROCYSBI 25 mg hard capsule
Light blue size 3 hard capsules imprinted “25 mg” in white ink and a light blue cap imprinted with 
“PRO” in white ink.

PROCYSBI 75 mg hard capsule
Light blue size 0 hard capsules imprinted “75 mg” in white ink and a dark blue cap imprinted with 
“PRO” in white ink.

4. CLINICAL PARTICULARS

4.1 Therapeutic indications

PROCYSBI is indicated for the treatment of proven nephropathic cystinosis. Cysteamine reduces 
cystine accumulation in some cells (e.g. leukocytes, muscle and liver cells) of nephropathic cystinosis 
patients and, when treatment is started early, it delays the development of renal failure. 

4.2 Posology and method of administration

PROCYSBI treatment should be initiated under the supervision of a physician experienced in the 
treatment of cystinosis.
Cysteamine therapy must be initiated promptly once the diagnosis is confirmed (i.e., increased WBC 
cystine) to achieve maximum benefit.

Posology

White blood cell (WBC) cystine concentration may for instance be measured by a number of different 
techniques such as specific WBC subsets (e.g., granulocyte assay) or the mixed leukocyte assay with 
each assay having different target values. Healthcare professionals should refer to the assay-specific 
therapeutic targets provided by individual testing laboratories when making decisions regarding 
diagnosis and PROCYSBI dosing for cystinosis patients. For example the therapeutic goal is to 
maintain a WBC cysteine level < 1 nmol hemicystine/mg protein (when measured using the mixed 
leukocyte assay), 30 min after dosing For patients adherent to a stable dose of PROCYSBI, and who 
do not have easy access to an adequate facility for measuring their WBC cystine, the goal of therapy 
should be to maintain plasma cysteamine concentration > 0.1 mg/L, 30 min after dosing.



3

Measurement timing: PROCYSBI should be administered every 12 hours. The determination of WBC 
cystine and/or plasma cysteamine must be obtained 12.5 hours after the evening dose the day before, 
and therefore 30 minutes after the following morning dose is given.

Transferring patients from immediate-release cysteamine bitartrate hard capsules 
Patients with cystinosis taking immediate-release cysteamine bitartrate may be transferred to a total 
daily dose of PROCYSBI equal to their previous total daily dose of immediate-release cysteamine 
bitartrate. Total daily dose should be divided by two and administered every 12 hours. The maximum 
recommended dose of cysteamine is 1.95 g/m2/day. The use of doses higher than 1.95 g/m2/day is not 
recommended (see section 4.4).
Patients being transferred from immediate-release cysteamine bitartrate to PROCYSBI should have 
their WBC cystine levels measured in 2 weeks, and thereafter every 3 months to assess optimal dose 
as described above.

Newly diagnosed adult patients
Newly diagnosed adult patients should be started on 1/6 to 1/4 of the targeted maintenance dose of 
PROCYSBI. The targeted maintenance dose is 1.3 g/m2/day, in two divided doses, given every 
12 hours. The dose should be raised if there is adequate tolerance and the WBC cystine level 
remains > 1 nmol hemicystine/mg protein (when measured using the mixed leukocyte assay). The 
maximum recommended dose of cysteamine is 1.95 g/m2/day. The use of doses higher than 
1.95 g/m2/day is not recommended (see section 4.4). 
The target values provided in the SmPC are obtained from using the mixed leucocyte assay. It should 
be noted that therapeutic targets for cystine depletion are assay-specific and different assays have 
specific treatment targets. Therefore, healthcare professionals should refer to the assay-specific 
therapeutic targets provided by individual testing laboratories.

Newly diagnosed paediatric population
The targeted maintenance dose of 1.3 g/m2/day can be approximated according to the following table, 
which takes surface area as well as weight into consideration. 

Weight in kilograms Recommended dose in mgEvery 12 hours*
0–5 200
5–10 300
11–15 400
16–20 500
21–25 600
26–30 700
31–40 800
41–50 900
> 50 1000

* Higher dose may be required to achieve target WBC cystine concentration. 
The use of doses higher than 1.95 g/m2/day is not recommended.

Special populations

Patients with poor tolerability
Patients with poorer tolerability still receive significant benefit if white blood cell cystine levels are 
below 2 nmol hemicystine/mg protein (when measured using the mixed leukocyte assay). The 
cysteamine dose can be increased to a maximum of 1.95 g/m2/day to achieve this level. The dose of 
1.95 g/m2/day of immediate-release cysteamine bitartrate has been associated with an increased rate of 
withdrawal from treatment due to intolerance and an increased incidence of adverse events. If 
cysteamine is initially poorly tolerated due to gastrointestinal (GI) tract symptoms or transient skin 
rashes, therapy should be temporarily stopped, then re-instituted at a lower dose and gradually 
increased to the appropriate dose (see section 4.4). 



4

Patients on dialysis or post-transplantation 
Experience has occasionally shown that some forms of cysteamine are less well tolerated (i.e. leading 
to more adverse events) when patients are on dialysis. A closer monitoring of the WBC cystine levels 
is recommended in these patients.  

Patients with renal impairment
Dose adjustment is not normally required; however, WBC cystine levels should be monitored.

Patients with hepatic impairment 
Dose adjustment is not normally required; however, WBC cystine levels should be monitored.

Method of administration

This medicinal product can be administered by swallowing the intact capsules as well as sprinkling the 
capsule contents (enteric coated beads) on food or delivery through a gastric feeding tube.
Do not crush or chew capsules or capsule contents.

Missed doses
If a dose is missed, it should be taken as soon as possible. If it is within four hours of the next dose, 
skip the missed dose and go back to the regular dosing schedule. Do not double the dose. 

Administration with food
Cysteamine bitartrate can be administered with an acidic fruit juice or water.
Cysteamine bitartrate should not be administered with food rich in fat or proteins, or with frozen food 
like ice-cream. Patients should try to consistently avoid meals and dairy products for at least 1 hour 
before and 1 hour after PROCYSBI dosing. If fasting during this period is not possible, it is acceptable 
to eat only a small amount ( 100 grams) of food (preferentially carbohydrates) during the hour before 
and after PROCYSBI administration. It is important to dose PROCYSBI in relation to food intake in a 
consistent and reproducible way over time (see section 5.2)
In paediatric patients who are at risk of aspiration, aged approximately 6 years and under, the hard 
capsules should be opened and the content sprinkled on food or liquid listed below.

Sprinkling on food
Capsules for either the morning or evening dose should be opened and the contents sprinkled onto 
approximately 100 grams of apple sauce or berry jelly. Gently stir the contents into the soft food, 
creating a mixture of cysteamine granules and food. The entire amount of the mixture should be eaten. 
This may be followed by 250 mL of an acceptable acidic liquid - fruit juice (e.g., orange juice or any 
acidic fruit juice) or water. The mixture must be eaten within 2 hours after preparation and must be 
refrigerated from the time of preparation to the time of administration.

Administering through feeding tubes
Capsules for either the morning or evening dose should be opened and the contents sprinkled onto 
approximately 100 grams of apple sauce or berry jelly. Gently stir the contents into the soft food, 
creating a mixture of cysteamine granules and the soft food. The mixture should then be administered 
via gastrostomy tube, nasogastric tube or gastrostomy-jejunostomy tube. The mixture must be 
administered within 2 hours after preparation and may be refrigerated from the time of preparation to 
the time of administration. 

Sprinkling in orange juice or any acidic fruit juice or water
Capsules for either the morning or evening dose should be opened and the contents sprinkled into 100 
to 150 mL of acidic fruit juice or water. Dose administration options are provided below: 
 Option 1 / Syringe: Mix gently for 5 minutes, then aspirate the mixture of cysteamine granules 

and acidic fruit juice or water into a dosing syringe.
 Option 2 / Cup: Mix gently for 5 minutes in a cup or shake gently for 5 minutes in a covered 

cup (e.g., “sippy” cup). Drink the mixture of cysteamine granules and acidic fruit juice or water.
The mixture must be administered (drunk) within 30 minutes after preparation and must be 
refrigerated from the time of preparation to the time of administration.



5

4.3 Contraindications

 Hypersensitivity to the active substance, any form of cysteamine (mercaptamine), or to any of 
the excipients listed in section 6.1.

 Hypersensitivity to penicillamine.
 Breast-feeding.

4.4 Special warnings and precautions for use

The use of doses higher than 1.95 g/m2/day is not recommended (see section 4.2).

Oral cysteamine has not been shown to prevent eye deposition of cystine crystals. Therefore, where 
cysteamine ophthalmic solution is used for that purpose, its usage should continue. 

If a pregnancy is diagnosed or planned, the treatment should be carefully reconsidered and the patient 
must be advised of the possible teratogenic risk of cysteamine (see section 4.6).

Intact capsules of PROCYSBI should not be administered to children under the age of approximately 
6 years due to risk of aspiration (see section 4.2).

Dermatological

There have been reports of serious skin lesions in patients treated with high doses of immediate-
release cysteamine bitartrate or other cysteamine salts that have responded to cysteamine dose 
reduction. Physicians should routinely monitor the skin and bones of patients receiving cysteamine.

If skin or bone abnormalities appear, the dose of cysteamine should be reduced or stopped. Treatment 
may be restarted at a lower dose under close supervision, and then slowly titrated to the appropriate 
therapeutic dose (see sections 4.2). If a severe skin rash develops such as erythema multiforme bullosa 
or toxic epidermal necrolysis, cysteamine should not be re-administered (see sections 4.8).

Gastrointestinal

GI ulceration and bleeding have been reported in patients receiving immediate-release cysteamine 
bitartrate. Physicians should remain alert for signs of ulceration and bleeding and should inform 
patients and/or guardians about the signs and symptoms of serious GI toxicity and what steps to take if 
they occur.

GI tract symptoms including nausea, vomiting, anorexia and abdominal pain have been associated 
with cysteamine.

Strictures of the ileo-caecum and large bowel (fibrosing colonopathy) was first described in cystic 
fibrosis patients who were given high doses of pancreatic enzymes in the form of tablets with an 
enteric coating of methacrylic acid - ethyl acrylate copolymer (1:1), one of the excipients in 
PROCYSBI. As a precaution, unusual abdominal symptoms or changes in abdominal symptoms 
should be medically assessed to exclude the possibility of fibrosing colonopathy.

Central Nervous System (CNS)

CNS symptoms such as seizures, lethargy, somnolence, depression, and encephalopathy have been 
associated with cysteamine. If CNS symptoms develop, the patient should be carefully evaluated and 
the dose adjusted as necessary. Patients should not engage in potentially hazardous activities until the 
effects of cysteamine on mental performance are known (see section 4.7). 



6

Leukopenia and abnormal liver function

Cysteamine has occasionally been associated with reversible leukopenia and abnormal liver function. 
Therefore, blood counts and liver function should be monitored. 

Benign intracranial hypertension

There have been reports of benign intracranial hypertension (or pseudotumor cerebri (PTC)) and/or 
papilledema associated with cysteamine bitartrate treatment that has resolved with the addition of 
diuretic therapy (post-marketing experience with the immediate-release cysteamine bitartrate). 
Physicians should instruct patients to report any of the following symptoms: headache, tinnitus, 
dizziness, nausea, diplopia, blurred vision, loss of vision, pain behind the eye or pain with eye 
movement. A periodic eye examination is needed to identify this condition early and timely treatment 
should be provided when it occurs to prevent vision loss. 

Important information about some of the excipients of PROCYSBI
This medicinal product contains less than 1 mmol sodium (23 mg) per dose, i.e. essentially sodium-
free.

4.5 Interaction with other medicinal products and other forms of interaction

It cannot be excluded that cysteamine is a clinically relevant inducer of CYP enzymes, inhibitor of 
P-gp and BCRP at the intestinal level and inhibitor of liver uptake transporters (OATP1B1, OATP1B3 
and OCT1).

Co-administration with electrolyte and mineral replacement

Cysteamine can be administered with electrolyte (except bicarbonate) and mineral replacements 
necessary for management of Fanconi syndrome as well as vitamin D and thyroid hormone. 
Bicarbonate should be administered at least one hour before or one hour after PROCYSBI to avoid 
potential earlier release of cysteamine.

Indomethacin and cysteamine have been used simultaneously in some patients. In cases of patients 
with kidney transplants, anti-rejection treatments have been used with cysteamine.

Co-administration of the proton pump inhibitor omeprazole and PROCYSBI in vivo showed no effects 
on cysteamine bitartrate exposure. 

4.6 Fertility, pregnancy and lactation

Pregnancy

There is no adequate data from the use of cysteamine in pregnant women. Studies in animals have 
shown reproductive toxicity, including teratogenesis (see section 5.3). The potential risk for humans is 
unknown. The effect on pregnancy of untreated cystinosis is also unknown. Therefore, cysteamine 
bitartrate should not be used during pregnancy, particularly during the first trimester, unless clearly 
necessary (see section 4.4).

If a pregnancy is diagnosed or planned, the treatment should be carefully reconsidered and the patient 
must be advised of the possible teratogenic risk of cysteamine.

Breast-feeding

Cysteamine excretion in human milk is unknown. However, due to the results of animal studies in 
breast-feeding females and neonates (see section 5.3), breast-feeding is contra-indicated in women 
taking PROCYSBI (see section 4.3).



7

Fertility

Effects on fertility have been seen in animal studies (see section 5.3). Azoospermia has been reported 
in male cystinosis patients.

4.7 Effects on ability to drive and use machines

Cysteamine has minor or moderate influence on the ability to drive and use machines.

Cysteamine may cause drowsiness. When starting therapy, patients should not engage in potentially 
hazardous activities until the effects of the medicinal product on each individual are known.

4.8 Undesirable effects 

Summary of the safety profile

For the immediate-release formulation of cysteamine bitartrate, approximately 35% of patients can be 
expected to experience adverse reactions. These mainly involve the gastrointestinal and central 
nervous systems. When these reactions appear at the initiation of cysteamine therapy, temporary 
suspension and gradual reintroduction of treatment may be effective in improving tolerance.
In clinical studies with healthy volunteers, the most frequent adverse reactions were very common GI 
symptoms (16%) and occurred primarily as single episodes that were mild or moderate in severity. 
The adverse reactions profile for healthy subjects was similar to the adverse reactions profile in 
patients relative to GI disorders (diarrhoea and abdominal pain). 

Tabulated list of adverse reactions

The frequency of adverse reactions is defined using the following convention: very common (≥1/10); 
common (≥1/100 to <1/10); uncommon (≥1/1,000 to <1/100); rare (≥1/10,000 to <1/1,000); very rare 
(<1/10,000) and not known (cannot be estimated from available data).
Within each frequency grouping, adverse reactions are presented in order of decreasing seriousness:

MedDRA system organ class Frequency: adverse reaction
Blood and lymphatic system 
disorders Uncommon: Leukopenia

Immune system disorders Uncommon: Anaphylactic reaction

Metabolism and nutrition disorders Very common: Anorexia

Psychiatric disorders Uncommon: Nervousness, hallucination

Common: Headache, encephalopathyNervous system disorders

Uncommon: Somnolence, convulsions
Very common: Vomiting, nausea, diarrhoea

Common: Abdominal pain, breath odour, dyspepsia, 
gastroenteritis

Gastrointestinal disorders

Uncommon: Gastrointestinal ulcer
Common: Skin odour abnormal, rashSkin and subcutaneous tissue 

disorders
Uncommon: Hair colour changes, skin striae, skin 
fragility (molluscoid pseudotumour on elbows)

Musculoskeletal and connective 
tissue disorders

Uncommon: Joint hyperextension, leg pain, genu 
valgum, osteopenia, compression fracture, scoliosis.



8

Renal and urinary disorders Uncommon: Nephrotic syndrome

Very common: Lethargy, pyrexiaGeneral disorders and 
administration site conditions Common: Asthenia
Investigations Common: Liver function tests abnormal

Description of selected adverse reactions

Clinical studies experience with PROCYSBI
In clinical studies comparing PROCYSBI to the immediate-release cysteamine bitartrate, one third of 
the patients exhibited very common GI disorders (nausea, vomiting, abdominal pain). Common 
nervous system disorders (headache, somnolence and lethargy) and common general disorders 
(asthenia) were also seen. 

Post-marketing experience with immediate-release cysteamine bitartrate
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 with immediate-release cysteamine 
bitartrate (see section 4.4). 

Two cases of nephrotic syndrome have been reported within 6 months of starting therapy with 
progressive recovery after treatment discontinuation. Histology showed a membranous 
glomerulonephritis of the renal allograft in one case and hypersensitivity interstitial nephritis in the 
other.

A few cases of Ehlers-Danlos-like syndrome on elbows have been reported in children chronically 
treated with high doses of different cysteamine preparations (cysteamine chlorhydrate or cystamine or 
cysteamine bitartrate) mostly above the maximal dose 1.95 g/m2/day. In some cases, these skin lesions 
were associated with skin striae and bone lesions first seen during an X-ray examination. Bone 
disorders reported were genu valgum, leg pain and hyperextensive joints, osteopenia, compression 
fractures, and scoliosis. In the few cases where histopathological examination of the skin was 
performed, the results suggested angioendotheliomatosis. One patient subsequently died of acute 
cerebral ischemia with marked vasculopathy. In some patients, the skin lesions on elbows regressed 
after immediate-release cysteamine dose reduction (see section 4.4).

Reporting of suspected adverse reactions 

Reporting suspected adverse reactions after authorisation of the medicinal product is important. It 
allows continued monitoring of the benefit/risk balance of the medicinal product. Healthcare 
professionals are asked to report any suspected adverse reactions via the national reporting system 
listed in Appendix V.

4.9 Overdose

An overdose of cysteamine may cause progressive lethargy.

Should overdosing occur, the respiratory and cardiovascular systems should be supported 
appropriately. No specific antidote is known. It is not known if cysteamine is removed by 
haemodialysis.



9

5. PHARMACOLOGICAL PROPERTIES

5.1 Pharmacodynamic properties

Pharmacotherapeutic group: Other alimentary tract and metabolism product, ATC code: A16AA04.

Cysteamine is the simplest stable aminothiol and a degradation product of the amino acid cysteine. 
Cysteamine participates within lysosomes in a thiol-disulfide interchange reaction converting cystine 
into cysteine and cysteine-cysteamine mixed disulfide, both of which can exit the lysosome in patients 
with cystinosis.

Normal individuals and persons heterozygous for cystinosis have white blood cell cystine levels of 
< 0.2 and usually below 1 nmol hemicystine/mg protein, respectively, when measured using the mixed 
leukocyte assay. Individuals with cystinosis have elevations of WBC cystine above 
2 nmol hemicystine/mg protein. 

WBC cystine is monitored in these patients to determine adequacy of dosing, levels being measured 
30 minutes after dosing when treated with PROCYSBI. 

A pivotal phase 3 randomized, crossover PK and PD study (which was also the first ever randomized 
study with immediate-release cysteamine bitartrate) demonstrated that at steady-state, patients 
receiving PROCYSBI every 12 hours (Q12H) maintained a comparable depletion of WBC cystine 
levels compared to immediate-release cysteamine bitartrate every 6 hours (Q6H). Forty-three (43) 
patients were randomized; twenty-seven (27) children (ages 6 to 12 years old), fifteen (15) adolescents 
(ages 12 to 21 years old) and one (1) adult with cystinosis and with native kidney function based on an 
estimated Glomerular Filtration Rate (GFR) (corrected for body surface area) > 30 mL/minute/1.73 m2 
were randomized. Of those forty-three (43) patients, two (2) siblings withdrew at the end of the first 
crossover period, due to a prior planned surgery in one (1) of them; forty-one (41) patients completed 
the protocol. Two (2) patients were excluded from the per-protocol analysis because their WBC 
cystine level increased over 2 nmol hemicystine/mg protein during the immediate-release cysteamine 
treatment period. Thirty-nine (39) patients were included in the final primary per protocol efficacy 
analysis. 

Per –Protocol (PP) Population (N=39)
Immediate-release 

cysteamine bitartrate PROCYSBI

WBC cystine level 
(LS Mean ± SE) in nmol hemicystine/mg 
protein*

0.44 ± 0.05 0.51 ± 0.05

Treatment effect
(LS mean ± SE; 95.8% CI; p-value) 0.08 ± 0.03; 0.01 to 0.15; <0.0001

All Evaluable Patients (ITT) Population (N=41)
Immediate-release 

cysteamine bitartrate PROCYSBI

WBC cystine level 
(LS Mean ± SE) in nmol hemicystine/mg 
protein*

0.74 ± 0.14 0.53 ± 0.14

Treatment effect 
(LS mean ± SE; 95.8% CI; p-value) -0.21 ± 0.14; -0.48 to 0.06; <0.001

* measured using the mixed leukocyte assay

Forty of forty-one (40/41) patients who completed the pivotal phase 3 study were entered in a 
prospective study with PROCYSBI that stayed open as long as PROCYSBI could not be prescribed by 
their treating physician. In this study, the WBC cystine measured using the mixed leukocyte assay was 
always on average under optimal control at < 1 nmol hemicystine/mg protein. The estimated 
glomerular filtration rate (eGFR) did not change for the study population over time. 



10

5.2 Pharmacokinetic properties

Absorption

The relative bioavailability is about 125% as compared to immediate-release cysteamine.

Food intake reduces the absorption of PROCYSBI at 30 minutes pre-dose (approximately 35% 
decrease in exposure) and at 30 min post-dose (approximately 16 or 45% decrease in exposure for 
intact and open capsules respectively). Food intake two hours after administration did not affect the 
absorption of PROCYSBI. 

Distribution

The in vitro plasma protein binding of cysteamine, primarily to albumin, is approximately 54% and 
independent of plasma drug concentration over the therapeutic range. 

Biotransformation

The elimination of unchanged cysteamine in the urine has been shown to range between 0.3% and 
1.7% of the total daily dose in four patients; the bulk of cysteamine is excreted as sulphate.

In vitro data suggests that cysteamine bitartrate is likely to be metabolized by multiple CYP enzymes, 
including CYP1A2, CYP2B6, CYP2C8, CYP2C9, CYP2C19, CYP2D6, and CYP2E1. CYP2A6 and 
CYP3A4 were not involved in the metabolism of cysteamine bitartrate under the experimental 
conditions. 

Elimination

The terminal half-life of cysteamine bitartrate is approximately 4 hours. 

Cysteamine bitartrate is not an inhibitor of CYP1A2, CYP2A6, CYP2B6, CYP2C8, CYP2C9, 
CYP2C19, CYP2D6, CYP2E1 and CYP3A4 in vitro.

In vitro: Cysteamine bitartrate is a substrate of P-gp and OCT2, but not a substrate of BCRP, 
OATP1B1, OATP1B3, OAT1, OAT3 and OCT1. Cysteamine bitartrate is not an inhibitor of OAT1, 
OAT3 and OCT2. 

Special populations

The pharmacokinetics of cysteamine bitartrate has not been studied in special populations. 

5.3 Preclinical safety data

In genotoxicity studies published for cysteamine, induction of chromosome aberrations in cultured 
eukaryotic cell lines has been reported. Specific studies with cysteamine did not show any mutagenic 
effects in the Ames test or any clastogenic effect in the mouse micronucleus test. A bacterial reverse 
mutation assay study (“Ames test”) was performed with the cysteamine bitartrate used for PROCYSBI 
and cysteamine bitartrate did not show any mutagenic effects in this test.

Reproduction studies showed embryo-foetotoxic effects (resorptions and post-implantation losses) in 
rats at the 100 mg/kg/day dose level and in rabbits receiving cysteamine 50 mg/kg/day. Teratogenic 
effects have been described in rats when cysteamine is administered over the period of organogenesis 
at a dose of 100 mg/kg/day.

This is equivalent to 0.6 g/m2/day in the rat, which is slightly less than the recommended clinical 
maintenance dose of cysteamine, i.e. 1.3 g/m2/day. A reduction of fertility was observed in rats at 
375 mg/kg/day, a dose at which body weight gain was retarded. At this dose, weight gain and survival 



11

of the offspring during lactation was also reduced. High doses of cysteamine impair the ability of 
lactating mothers to feed their pups. Single doses of the drug inhibit prolactin secretion in animals.

Administration of cysteamine in neonate rats induced cataracts.

High doses of cysteamine, either by oral or parenteral routes, produce duodenal ulcers in rats and mice 
but not in monkeys. Experimental administration of the drug causes depletion of somatostatin in 
several animal species. The consequence of this for the clinical use of the drug is unknown.

No carcinogenic studies have been conducted with cysteamine bitartrate gastro-resistant hard capsules.

6. PHARMACEUTICAL PARTICULARS

6.1 List of excipients

Capsule content 

microcrystalline cellulose
methacrylic acid - ethyl acrylate copolymer (1:1)
hypromellose
talc
triethyl citrate
sodium lauryl sulphate

Capsule shell 

gelatin
titanium dioxide (E171)
indigo carmine (E132)

Printing ink 

shellac
povidone K-17
titanium dioxide (E171)

6.2 Incompatibilities

Not applicable.

6.3 Shelf life

24 months
In-use shelf life: 30 days.

6.4 Special precautions for storage

Store in a refrigerator (2°C-8°C). Do not freeze.
After opening do not store above 25°C.
Keep the container tightly closed in order to protect from light and moisture.

6.5 Nature and contents of container

PROCYSBI 25 mg hard capsule
50 mL white HDPE bottle containing 60 capsules with one 2-in-1 desiccant cylinder and one oxygen 
absorber cylinder, with a child resistant polypropylene closure.



12

Each bottle contains two plastic cylinders used for additional moisture and air protection.
Please keep the two cylinders in each bottle during the use of the bottle. The cylinders may be 
discarded with the bottle after use.

PROCYSBI 75 mg hard capsule
400 mL white HDPE bottle containing 250 capsules with one 2-in-1 desiccant cylinder and two 
oxygen absorber cylinders, with a child resistant polypropylene closure.
Each bottle contains three plastic cylinders used for additional moisture and air protection.
Please keep the three cylinders in each bottle during the use of the bottle. The cylinders may be 
discarded with the bottle after use.

6.6 Special precautions for disposal

No special requirements.

7. MARKETING AUTHORISATION HOLDER

Chiesi Farmaceutici S.p.A.
Via Palermo 26/A
43122 Parma 
Italy

8. MARKETING AUTHORISATION NUMBER(S)

PROCYSBI 25 mg hard capsule
EU/1/13/861/001

PROCYSBI 75 mg hard capsule
EU/1/13/861/002

9. DATE OF FIRST AUTHORISATION/RENEWAL OF THE AUTHORISATION

Date of first authorisation: 06 September 2013
Date of latest renewal: 26 July 2018

10. DATE OF REVISION OF THE TEXT

Detailed information on this medicinal product is available on the website of the European Medicines 
Agency http://www.ema.europa.eu.



13

ANNEX II

A. MANUFACTURER RESPONSIBLE FOR BATCH RELEASE

B. CONDITIONS OR RESTRICTIONS REGARDING SUPPLY 
AND USE

C. OTHER CONDITIONS AND REQUIREMENTS OF THE 
MARKETING AUTHORISATION

D. CONDITIONS OR RESTRICTIONS WITH REGARD TO 
THE SAFE AND EFFECTIVE USE OF THE MEDICINAL 
PRODUCT



14

A. MANUFACTURER RESPONSIBLE FOR BATCH RELEASE

Name and address of the manufacturer responsible for batch release

Chiesi Farmaceutici S.p.A.
Via San Leonardo 96
43122 Parma 
Italy

B. CONDITIONS OR RESTRICTIONS REGARDING SUPPLY AND USE 

Medicinal product subject to restricted medical prescription (see Annex I: Summary of Product 
Characteristics, section 4.2).

C. OTHER CONDITIONS AND REQUIREMENTS OF THE MARKETING 
AUTHORISATION

 Periodic Safety Update Reports

The requirements for submission of periodic safety update reports for this medicinal product are set 
out in the list of Union reference dates (EURD list) provided for under Article 107c(7) of Directive 
2001/83/EC and any subsequent updates published on the European medicines web-portal. 

D. CONDITIONS OR RESTRICTIONS WITH REGARD TO THE SAFE AND 
EFFECTIVE USE OF THE MEDICINAL PRODUCT  

 Risk Management Plan (RMP)

The MAH shall perform the required pharmacovigilance activities and interventions detailed in the 
agreed RMP presented in Module 1.8.2 of the Marketing Authorisation and any agreed subsequent 
updates of the RMP.

An updated RMP should be submitted:
 At the request of the European Medicines Agency;
 Whenever the risk management system is modified, especially as the result of new information 

being received that may lead to a significant change to the benefit/risk profile or as the result of 
an important (pharmacovigilance or risk minimisation) milestone being reached.

 Additional risk minimisation measures 

The MAH shall provide an educational pack targeting all physicians who are expected to 
prescribe PROCYSBI prior to the launch.
The education pack is aimed at strengthening awareness of important identified and potential 
risks as well as appropriate patient selection, the need for dose titration and patient 
monitoring. 

The physician education pack should contain the Safety Checklist, the Summary of Product 
Characteristics and Package Leaflet.

The Safety Checklist should highlight the following:
 The risk of teratogenicity and relevant risk minimisation advice:
 Women of childbearing potential should be informed about the risk of teratogenicity;
 For women of child-bearing potential a negative pregnancy test should be confirmed 

before starting treatment;



15

 Women of child-bearing potential should be advised to use an adequate method of 
contraception during the course of treatment;

 Women of child-bearing potential should be advised to alert the treating physician if 
they become pregnant during treatment.

 The risk of fibrosing colonopathy and relevant risk minimisation advice:
 Patients should be informed about the potential risk of fibrosing colonopathy;
 Patients should be advised of the signs and symptoms of fibrosing colonopathy and to 

alert the treating physician if they develop any.
 Guidance on appropriate patient selection and dose titration.
 The need for monitoring of white blood cell cystine levels, full blood count and liver 

function.
 The need to monitor regularly skin and to consider X-ray examinations of the bone as 

necessary.
 The need to advise patients about:
 The method of administration and timing of medicine intake 
 The need to contact the treating physician if they experience the following events:
 Problems or changes with their skin
Upset in their normal bowel habit, 
 Lethargy, somnolence depression, fits
Any suspicion that they might be pregnant

The MAH must agree the content and format of the educational material, together with a 
communication plan, with the national competent authority prior to distribution of the educational 
pack.



16

ANNEX III

LABELLING AND PACKAGE LEAFLET



17

A. LABELLING



18

PARTICULARS TO APPEAR ON THE OUTER PACKAGING

OUTER CARTON

1. NAME OF THE MEDICINAL PRODUCT

PROCYSBI 25 mg gastro-resistant hard capsules
cysteamine

2. STATEMENT OF ACTIVE SUBSTANCE(S)

Each capsule contains 25 mg of cysteamine (as mercaptamine bitartrate).

3. LIST OF EXCIPIENTS

4. PHARMACEUTICAL FORM AND CONTENTS

Gastro-resistant hard capsule

60 capsules

5. METHOD AND ROUTE(S) OF ADMINISTRATION

Read the package leaflet before use.
Oral use.

6. SPECIAL WARNING THAT THE MEDICINAL PRODUCT MUST BE STORED OUT 
OF THE SIGHT AND REACH OF CHILDREN

Keep out of the sight and reach of children.

7. OTHER SPECIAL WARNING(S), IF NECESSARY

8. EXPIRY DATE

EXP

Discard 30 days after opening the foil seal.

9. SPECIAL STORAGE CONDITIONS

Store in a refrigerator. Do not freeze.
After opening do not store above 25°C.
Keep the container tightly closed in order to protect from light and moisture.



19

10. SPECIAL PRECAUTIONS FOR DISPOSAL OF UNUSED MEDICINAL PRODUCTS 
OR WASTE MATERIALS DERIVED FROM SUCH MEDICINAL PRODUCTS, IF 
APPROPRIATE

11. NAME AND ADDRESS OF THE MARKETING AUTHORISATION HOLDER

Chiesi Farmaceutici S.p.A.
Via Palermo 26/A
43122 Parma
Italy

12. MARKETING AUTHORISATION NUMBER(S) 

EU/1/13/861/001

13. BATCH NUMBER

Lot

14. GENERAL CLASSIFICATION FOR SUPPLY

15. INSTRUCTIONS ON USE

16. INFORMATION IN BRAILLE

PROCYSBI 25 mg

17. UNIQUE IDENTIFIER – 2D BARCODE

2D barcode carrying the unique identifier included.

18. UNIQUE IDENTIFIER - HUMAN READABLE DATA

PC: 
SN: 
NN: 



20

PARTICULARS TO APPEAR ON THE OUTER PACKAGING

OUTER CARTON 

1. NAME OF THE MEDICINAL PRODUCT

PROCYSBI 75 mg gastro-resistant hard capsules
cysteamine

2. STATEMENT OF ACTIVE SUBSTANCE(S)

Each capsule contains 75 mg of cysteamine (as mercaptamine bitartrate).

3. LIST OF EXCIPIENTS

4. PHARMACEUTICAL FORM AND CONTENTS

Gastro-resistant hard capsule

250 capsules

5. METHOD AND ROUTE(S) OF ADMINISTRATION

Read the package leaflet before use.
Oral use.

6. SPECIAL WARNING THAT THE MEDICINAL PRODUCT MUST BE STORED OUT 
OF THE SIGHT AND REACH OF CHILDREN

Keep out of the sight and reach of children.

7. OTHER SPECIAL WARNING(S), IF NECESSARY

8. EXPIRY DATE

EXP

Discard 30 days after opening the foil seal.

9. SPECIAL STORAGE CONDITIONS

Store in a refrigerator. Do not freeze.
After opening do not store above 25°C.
Keep the container tightly closed in order to protect from light and moisture.



21

10. SPECIAL PRECAUTIONS FOR DISPOSAL OF UNUSED MEDICINAL PRODUCTS 
OR WASTE MATERIALS DERIVED FROM SUCH MEDICINAL PRODUCTS, IF 
APPROPRIATE

11. NAME AND ADDRESS OF THE MARKETING AUTHORISATION HOLDER

Chiesi Farmaceutici S.p.A.
Via Palermo 26/A
43122 Parma
Italy

12. MARKETING AUTHORISATION NUMBER(S) 

EU/1/13/861/002

13. BATCH NUMBER

Lot

14. GENERAL CLASSIFICATION FOR SUPPLY

15. INSTRUCTIONS ON USE

16. INFORMATION IN BRAILLE

PROCYSBI 75 mg

17. UNIQUE IDENTIFIER – 2D BARCODE

2D barcode carrying the unique identifier included.

18. UNIQUE IDENTIFIER - HUMAN READABLE DATA

PC: 
SN: 
NN: 



22

PARTICULARS TO APPEAR ON THE IMMEDIATE PACKAGING

BOTTLE 

1. NAME OF THE MEDICINAL PRODUCT

PROCYSBI 25 mg gastro-resistant hard capsules
cysteamine

2. STATEMENT OF ACTIVE SUBSTANCE(S)

Each capsule contains 25 mg of cysteamine (as mercaptamine bitartrate).

3. LIST OF EXCIPIENTS

4. PHARMACEUTICAL FORM AND CONTENTS

Gastro-resistant hard capsule

60 capsules

5. METHOD AND ROUTE(S) OF ADMINISTRATION

Read the package leaflet before use.
Oral use.

6. SPECIAL WARNING THAT THE MEDICINAL PRODUCT MUST BE STORED OUT 
OF THE SIGHT AND REACH OF CHILDREN

Keep out of the sight and reach of children.

7. OTHER SPECIAL WARNING(S), IF NECESSARY

8. EXPIRY DATE

EXP

Discard 30 days after opening the foil seal.
Open Date:
Discard Date:

9. SPECIAL STORAGE CONDITIONS

Store in a refrigerator. Do not freeze.
After opening do not store above 25°C.



23

Keep the container tightly closed in order to protect from light and moisture.

10. SPECIAL PRECAUTIONS FOR DISPOSAL OF UNUSED MEDICINAL PRODUCTS 
OR WASTE MATERIALS DERIVED FROM SUCH MEDICINAL PRODUCTS, IF 
APPROPRIATE

11. NAME AND ADDRESS OF THE MARKETING AUTHORISATION HOLDER

Chiesi Farmaceutici S.p.A.
Via Palermo 26/A
43122 Parma
Italy

12. MARKETING AUTHORISATION NUMBER(S) 

EU/1/13/861/001

13. BATCH NUMBER

Lot

14. GENERAL CLASSIFICATION FOR SUPPLY

15. INSTRUCTIONS ON USE

16. INFORMATION IN BRAILLE

17. UNIQUE IDENTIFIER – 2D BARCODE

2D barcode carrying the unique identifier included.

18. UNIQUE IDENTIFIER - HUMAN READABLE DATA

PC: 
SN: 
NN: 



24

PARTICULARS TO APPEAR ON THE IMMEDIATE PACKAGING

BOTTLE 

1. NAME OF THE MEDICINAL PRODUCT

PROCYSBI 75 mg gastro-resistant hard capsules 
cysteamine

2. STATEMENT OF ACTIVE SUBSTANCE(S)

Each capsule contains 75 mg of cysteamine (as mercaptamine bitartrate).

3. LIST OF EXCIPIENTS

4. PHARMACEUTICAL FORM AND CONTENTS

Gastro-resistant hard capsule

250 capsules

5. METHOD AND ROUTE(S) OF ADMINISTRATION

Read the package leaflet before use.
Oral use.

6. SPECIAL WARNING THAT THE MEDICINAL PRODUCT MUST BE STORED OUT 
OF THE SIGHT AND REACH OF CHILDREN

Keep out of the sight and reach of children.

7. OTHER SPECIAL WARNING(S), IF NECESSARY

8. EXPIRY DATE

EXP

Discard 30 days after opening the foil seal.
Open Date:
Discard Date:

9. SPECIAL STORAGE CONDITIONS

Store in a refrigerator. Do not freeze.
After opening do not store above 25°C.
Keep the container tightly closed in order to protect from light and moisture.



25

10. SPECIAL PRECAUTIONS FOR DISPOSAL OF UNUSED MEDICINAL PRODUCTS 
OR WASTE MATERIALS DERIVED FROM SUCH MEDICINAL PRODUCTS, IF 
APPROPRIATE

11. NAME AND ADDRESS OF THE MARKETING AUTHORISATION HOLDER

Chiesi Farmaceutici S.p.A.
Via Palermo 26/A
43122 Parma
Italy

12. MARKETING AUTHORISATION NUMBER(S)

EU/1/13/861/002

13. BATCH NUMBER

Lot

14. GENERAL CLASSIFICATION FOR SUPPLY

15. INSTRUCTIONS ON USE

16. INFORMATION IN BRAILLE

17. UNIQUE IDENTIFIER – 2D BARCODE

2D barcode carrying the unique identifier included.

18. UNIQUE IDENTIFIER - HUMAN READABLE DATA

PC: 
SN: 
NN: 



26

B. PACKAGE LEAFLET



27

Package leaflet: Information for the user

PROCYSBI 25 mg gastro-resistant hard capsules
PROCYSBI 75 mg gastro-resistant hard capsules

Cysteamine (mercaptamine bitartrate)

Read all of this leaflet carefully before you start using this medicine because it contains 
important information for you.
- Keep this leaflet. You may need to read it again.
- If you have further questions, ask your doctor or pharmacist. 
- This medicine has been prescribed for you only. Do not pass it on to others. It may harm them, 

even if their signs of illness are the same as yours. 
- If you get any side effects, talk to your doctor or pharmacist. This includes any possible side 

effects not listed in this leaflet. See section 4.

What is in this leaflet

1. What PROCYSBI is and what it is used for
2. What you need to know before you take PROCYSBI 
3. How to take PROCYSBI 
4. Possible side effects 
5. How to store PROCYSBI
6. Contents of the pack and other information

1. What PROCYSBI is and what it is used for

PROCYSBI contains the active substance cysteamine (also known as mercaptamine) and is taken for 
the treatment of nephropathic cystinosis in children and adults. Cystinosis is a disease affecting how 
the body functions, with an abnormal accumulation of the amino acid cystine in various organs of the 
body such as the kidney, eye, muscle, pancreas, and brain. Cystine build-up causes kidney damage and 
excretion of excess amounts of glucose, proteins, and electrolytes. Different organs are affected at 
different ages.

PROCYSBI is a medicine that reacts with cystine to decrease its level within the cells. Cysteamine 
therapy should be initiated promptly after confirmation of the diagnosis of cystinosis to achieve 
maximum benefit.

2. What you need to know before you take PROCYSBI

Do not take PROCYSBI:
 If you are allergic to cysteamine (also known as mercaptamine) or any of the other ingredients 

of this medicine (listed in section 6).
 If you are allergic to penicillamine.
 If you are breast-feeding.

Warnings and precautions
Talk to your doctor or pharmacist before taking PROCYSBI.

 Since oral cysteamine doesn’t prevent deposits of cystine crystals in the eye, you should 
continue taking cysteamine eye drops as prescribed by your doctor. 

 Whole cysteamine capsules should not be given to children under the age of 6 years due to the 
risk of choking (refer to section 3 How to take PROCYSBI – Method of administration).



28

 Serious skin lesions can occur in patients treated with high doses of cysteamine. Your doctor 
will routinely monitor your skin and bones and reduce or stop your treatment if needed (see 
section 4).

 Stomach and intestinal ulcers and bleeding can occur in patients receiving cysteamine (see 
section 4). 

 Other intestinal symptoms including nausea, vomiting, anorexia and stomach ache can occur 
with cysteamine. Your doctor may interrupt and change your dose if these occur. 

 Talk to your doctor if you have any unusual stomach symptoms or changes in stomach 
symptoms.

 Symptoms such as seizures, tiredness, sleepiness, depression, and brain disorders 
(encephalopathy) can occur with cysteamine. If such symptoms develop, tell your doctor who 
will adjust your dose.

 Abnormal liver function or reduced white blood cell count (leukopenia) can occur with use of 
cysteamine. Your doctor will routinely monitor your blood counts and liver function.

 Your doctor will monitor you for benign intracranial hypertension (or pseudotumor cerebri 
(PTC)) and/or swelling of the optic nerve (papilledema) associated with cysteamine treatment. 
You will receive regular eye examinations to identify this condition as early treatment can 
prevent vision loss.

Other medicines and PROCYSBI
Tell your doctor or pharmacist if you are taking, have recently taken, or might take any other 
medicines. If your doctor prescribes bicarbonate, do not take it at the same time as PROCYSBI; take 
bicarbonate at least one hour before or at least one hour after the medicine.

PROCYSBI with food and drink
For at least 1 hour before and 1 hour after taking PROCYSBI try to avoid meals, which are rich in fat 
or proteins as well as any food or liquid that could decrease the acidity in your stomach, like milk or 
yogurt. If this is not possible, you can eat a small amount (about 100 grams) of food (preferably 
carbohydrates e.g. bread, pasta, fruits) during the hour before and after taking PROCYSBI. 
Take the capsule with an acidic drink (such as orange juice or any acidic juice) or water. For children 
and patients who have problems to swallow, please refer to section 3 How to take PROCYSBI – 
Method of administration.

Pregnancy and breastfeeding
If you are pregnant or breast-feeding, think you may be pregnant or are planning to have a baby, ask 
your doctor or pharmacist for advice before taking this medicine.

You should not use this medicine if you are pregnant, particularly during the first trimester. If you are 
a woman planning a pregnancy or become pregnant, seek immediate advice from your doctor about 
stopping therapy with this medicine as continued treatment may be harmful to the unborn baby. 

Do not use this medicine if you are breastfeeding (see section 2 under “Do not use PROCYSBI”). 

Driving and using machines
This medicine may cause some drowsiness. When starting therapy, you should not drive, use 
machines, or engage in other dangerous activities until you know how the medicine affects you.

PROCYSBI contains sodium
This medicine contains less than 1 mmol sodium (23 mg) per dose, that is to say essentially “sodium-
free.” 

3. How to take PROCYSBI

Always take this medicine exactly as your doctor or pharmacist has told you. Check with your doctor 
or pharmacist if you are not sure.



29

The recommended dose for you or your child will depend on your or your child’s age and weight. The 
targeted maintenance dose is 1.3 g/m2/day.

Dosing schedule
Take this medicine two times a day, every 12 hours. To get the most benefit from this medicine, try to 
avoid meals and dairy products for at least 1 hour before and 1 hour after PROCYSBI dosing. If this is 
not possible, you can eat a small amount (about 100 grams) of food (preferably carbohydrates e.g. 
bread, pasta, fruits) during the hour before and after PROCYSBI administration. 

It is important to take PROCYSBI in a consistent way over time.

Do not increase or decrease the amount of medicine without your doctor’s approval.

The total usual dose should not exceed 1.95 g/m2/day.

Duration of treatment
Treatment with PROCYSBI should continue life-long, as instructed by your doctor.

Method of administration
You should take this medicine only by mouth.

In order for this medicine to work correctly, you must do the following:
- Swallow the whole capsule with an acidic drink (such as orange juice or any acidic juice) or 

water. Do not crush or chew capsules or capsule contents. Do not give gastro-resistant hard 
capsules to children under 6 years of age because they may not be able to swallow them and 
they may choke. For patients who cannot swallow the whole capsule, the gastro-resistant hard 
capsule may be opened and the contents may be sprinkled on food (such as apple sauce or berry 
jelly) or mixed in with an acidic drink (such as orange juice or any acidic juice) or water. 
Consult your child’s doctor for complete directions.

- Your medical treatment may include, in addition to cysteamine, one or more supplements to 
replace important electrolytes lost through the kidneys. It is important to take these supplements 
exactly as instructed. If several doses of the supplements are missed or weakness or drowsiness 
develops, call your doctor for instructions.

- Regular blood tests to measure the amount of cystine inside white blood cells and/or the 
concentration of cysteamine in the blood are necessary to help determine the correct dose of 
PROCYSBI. You or your doctor will arrange for these blood tests to be performed. These tests 
must be obtained 12.5 hours after the evening dose the day before, and therefore 30 minutes 
after the following morning dose is given. Regular blood and urine tests to measure the levels of 
the body’s important electrolytes are also necessary to help you or your doctor correctly adjust 
the doses of these supplements.

If you take more PROCYSBI than you should
You should contact your doctor or the hospital emergency department immediately if you have taken 
more PROCYSBI than you should. You may become drowsy.

If you forget to take PROCYSBI
If you missed a dose of medicine, you should take it as soon as possible. However, if it is within 
4 hours of the next dose, skip the missed dose and go back to the regular dosing schedule. 

Do not take a double dose to make up for a forgotten dose.

If you have any further questions on the use of this medicine, ask your doctor or pharmacist.



30

4. Possible side effects 

Like all medicines, this medicine can cause side effects, although not everybody gets them. 

Tell your doctor or nurse straight away if you notice any of the following side effects – you may 
need urgent medical treatment:
 Severe allergic reaction (seen uncommonly): Get emergency medical help if you have any of 

these signs of an allergic reaction: hives; difficulty breathing; swelling of face, lips, tongue, or 
throat.

If any of the following side effects occur, please contact your doctor immediately. Since some of these 
side effects are serious, ask your doctor to explain their warning signs. 

Common side effects (may affect up to 1 in 10 people):
 Skin rash: Tell the doctor right away if you get a skin rash. PROCYSBI may need to be 

temporarily stopped until the rash goes away. If the rash is severe, your doctor may discontinue 
cysteamine treatment.

 Abnormal liver function on blood tests. Your doctor will monitor you for this.

Uncommon side effects (may affect up to 1 in 100 people):
 Skin lesions, bone lesions, and joint problems: Treatment with high doses of cysteamine can 

cause skin lesions to develop. These include skin striae (which are like stretch marks), bone 
injuries (such as fractures), bone deformities, and joint problems. Examine your skin while 
taking this medicine. Report any changes to your doctor. Your doctor will monitor you for these 
problems.

 Low white blood cell count. Your doctor will monitor you for this.
 Central nervous system symptoms: Some patients taking cysteamine have developed seizures, 

depression, and become too sleepy (excessive sleepiness). Tell your doctor if you have these 
symptoms.

 Stomach and intestinal (gastrointestinal) problems: Patients taking cysteamine have developed 
ulcers and bleeding. Tell your doctor right away if you get stomach ache, nausea, vomiting, loss 
of appetite, or throw up blood.

 Benign intracranial hypertension, also called pseudotumor cerebri, has been reported with 
cysteamine use. This is a condition where there is high pressure in the fluid around the brain. 
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. Your 
doctor will monitor you with eye examinations to find and treat this problem early. This will 
help lessen the chance of loss of eyesight.

The other side effects listed below are given with an estimation of the frequency with which they may 
occur with PROCYSBI.

Very common side effects (may affect more than 1 in 10 people):
 diarrhoea
 fever
 sensation of sleep

Common side effects:
 unpleasant breath and body odour
 heartburn
 tiredness

Uncommon side effects:
 leg pain
 scoliosis (deviation of the vertebral column)



31

 bone fragility
 hair discolouration
 fits
 nervousness
 hallucination
 effect on the kidney manifested by swelling of the extremities and weight gain

Reporting of side effects
If you get any side effects, talk to your doctor or pharmacist. This includes any possible side effects 
not listed in this leaflet. You can also report side effects directly via the national reporting system 
listed in Appendix V. By reporting side effects you can help provide more information on the safety of 
this medicine.

5. How to store PROCYSBI

Keep this medicine out of the sight and reach of children.

Do not use this medicine after the expiry date, which is stated on the carton and the bottle label after 
EXP. The expiry date refers to the last day of that month.

Do not take this medicine if the foil seal has been open for more than 30 days. Discard the open bottle 
and use a new bottle.

Store in a refrigerator (2°C-8°C). Do not freeze. After opening do not store above 25°C. Keep the 
container tightly closed in order to protect from light and moisture.

Do not throw away any medicines via wastewater. Ask your pharmacist how to throw away the 
medicines you no longer use. These measures will help protect the environment.

6. Contents of the pack and other information 

What PROCYSBI contains
 The active substance is cysteamine (as mercaptamine bitartrate). Each gastro-resistant hard 

capsule contains 25 mg or 75 mg of cysteamine.
 The other ingredients are:

o In the capsules: microcrystalline cellulose, methacrylic acid - ethyl acrylate copolymer 
(1:1), hypromellose, talc, triethyl citrate, sodium lauryl sulfate.

o In the capsule shell: gelatin, titanium dioxide (E171), indigo carmine (E132).
o In the printing ink: shellac, povidone (K-17), titanium dioxide (E171).

What PROCYSBI looks like and contents of the pack
 PROCYSBI 25 mg is presented as blue gastro-resistant hard capsules. The light blue cap is 

imprinted with “PRO” in white ink and the light blue body is imprinted with “25 mg” in white 
ink. A white plastic bottle contains 60 capsules. The cap is child resistant and has a foil seal. 
Each bottle contains two plastic cylinders used for additional moisture and air protection

 PROCYSBI 75 mg is presented as blue gastro-resistant hard capsules. The dark blue cap is 
imprinted with “PRO” in white ink and the light blue body is imprinted with “75 mg” in white 
ink. A white plastic bottle contains 250 capsules. The cap is child resistant and has a foil seal. 
Each bottle contains three plastic cylinders used for additional moisture and air protection. 

 Please keep the cylinders in each bottle during the use of the bottle. The cylinders may be 
discarded with the bottle after use.



32

Marketing Authorisation Holder 
Chiesi Farmaceutici S.p.A.
Via Palermo 26/A
43122 Parma
Italy

Manufacturer
Chiesi Farmaceutici S.p.A.
Via San Leonardo 96
43122 Parma 
Italy

For any information about this medicine, please contact the local representative of the Marketing 
Authorisation Holder:

België/Belgique/Belgien
Chiesi sa/nv 
Tél/Tel: + 32 (0)2 788 42 00

Lietuva
Chiesi Pharmaceuticals GmbH 
Tel: + 43 1 4073919

България
Chiesi Bulgaria EOOD 
Teл.: + 359 29201205

Luxembourg/Luxemburg
Chiesi sa/nv 
Tél/Tel: + 32 (0)2 788 42 00

Česká republika
Chiesi CZ s.r.o. 
Tel: + 420 261221745

Magyarország
Chiesi Hungary Kft. 
Tel.: + 36-1-429 1060

Danmark
Chiesi Pharma AB 
Tlf: + 46 8 753 35 20

Malta
Chiesi Farmaceutici S.p.A. 
Tel: + 39 0521 2791

Deutschland
Chiesi GmbH 
Tel: + 49 40 89724-0

Nederland
Chiesi Pharmaceuticals B.V. 
Tel: + 31 88 501 64 00

Eesti
Chiesi Pharmaceuticals GmbH 
Tel: + 43 1 4073919

Norge
Chiesi Pharma AB 
Tlf: + 46 8 753 35 20

Ελλάδα
Chiesi Hellas AEBE 
Τηλ: + 30 210 6179763

Österreich
Chiesi Pharmaceuticals GmbH 
Tel: + 43 1 4073919

España
Chiesi España, S.A.U. 
Tel: + 34 93 494 8000

Polska
Chiesi Poland Sp. z.o.o. 
Tel.: + 48 22 620 1421

France
Chiesi S.A.S. 
Tél: + 33 1 47688899

Portugal
Chiesi Farmaceutici S.p.A. 
Tel: + 39 0521 2791

Hrvatska
Chiesi Pharmaceuticals GmbH 
Tel: + 43 1 4073919

România
Chiesi Romania S.R.L. 
Tel: + 40 212023642



33

Ireland
Chiesi Farmaceutici S.p.A.  
Tel: + 39 0521 2791

Slovenija
Chiesi Slovenija d.o.o. 
Tel: + 386-1-43 00 901

Ísland
Chiesi Pharma AB 
Sími: +46 8 753 35 20

Slovenská republika
Chiesi Slovakia s.r.o. 
Tel: + 421 259300060

Italia
Chiesi Farmaceutici S.p.A. 
Tel: + 39 0521 2791

Suomi/Finland
Chiesi Pharma AB 
Puh/Tel: +46 8 753 35 20

Κύπρος
Chiesi Farmaceutici S.p.A. 
Τηλ: + 39 0521 2791

Sverige
Chiesi Pharma AB 
Tel: +46 8 753 35 20

Latvija
Chiesi Pharmaceuticals GmbH 
Tel: + 43 1 4073919

United Kingdom
Chiesi Ltd 
Tel: + 44 (0)161 488 5555

This leaflet was last revised in 

Detailed information on this medicine is available on the website of the European Medicines Agency 
http://www.ema.europa.eu.


	SUMMARY OF PRODUCT CHARACTERISTICS
	A.	MANUFACTURER RESPONSIBLE FOR BATCH RELEASE
	B.	CONDITIONS OR RESTRICTIONS REGARDING SUPPLY AND USE
	C.	OTHER CONDITIONS AND REQUIREMENTS OF THE MARKETING AUTHORISATION
	D.	CONDITIONS OR RESTRICTIONS WITH REGARD TO THE SAFE AND EFFECTIVE USE OF THE MEDICINAL PRODUCT
	A. LABELLING
	B. PACKAGE LEAFLET