Procysbi

mercaptamine

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This is a summary of the European public assessment report (EPAR) for Procysbi. It explains how the European Medicines Agency assessed the medicine to recommend its authorisation in the EU and its conditions of use. It is not intended to provide practical advice on how to use Procysbi.

For practical information about using Procysbi, patients should read the package leaflet or contact their doctor or pharmacist.

What is Procysbi and what is it used for?

Procysbi is a medicine that contains the active substance mercaptamine (also known as cysteamine). It is used in patients with nephropathic (kidney) cystinosis. Cystinosis is an inherited disease in which excess amounts of cystine, an amino acid naturally found in the body, build up within cells, especially in the kidneys and the eyes, damaging them.

Because the number of patients with cystinosis is low, the disease is considered ‘rare’, and Procysbi was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 20 September 2010.

Procysbi is a ‘hybrid medicine’. This means that it is similar to a ‘reference medicine’ containing the same active substance, but Procysbi is available in a formulation that allows for a delayed release of the active substance in the body. The reference medicine for Procysbi is Cystagon.

How is Procysbi used?

Procysbi can only be obtained with a prescription and treatment should be started under the supervision of a doctor who has experience in the treatment of cystinosis.

Procysbi is available as gastroresistant capsules (25 and 75 mg). Gastroresistant means that the capsules' contents pass through the stomach without being broken down until they reach the intestine. The recommended daily dose is calculated according to body surface area, as 1.30 g/m2 divided into two doses given every 12 hours. Cystine levels in white blood cells (which are measured as nanomoles of hemicystine per milligram of white-blood-cell protein), or alternatively mercaptamine concentration in the blood, should be monitored and used to adjust the dose, which should never exceed 1.95 g/m2 per day. For further information, see the package leaflet.

How does Procysbi work?

The active substance in Procysbi, mercaptamine, reacts with cystine to form another amino acid, called cysteine, and a compound called a cysteine-cysteamine salt. The body is able to remove this salt from the cells. The amount of cystine in the organs is therefore reduced, and this limits the damage to these organs.

What benefits of Procysbi have been shown in studies?

Procysbi given every 12 hours has been shown to be at least as effective as Cystagon given every six hours at maintaining the amount of cystine in white blood cells at acceptable levels (less than 1 nmol hemicystine per milligram of white-blood-cell protein). In a main study involving 43 patients with nephropathic cystinosis, there was no meaningful difference between the average levels of cystine in white blood cells during a three-week treatment with the two medicines. Levels were 0.51 nmol/mg with Procysbi, compared with 0.44 nmol/mg with Cystagon.

What are the risks associated with Procysbi?

The most common side effects with Procysbi (which may affect more than 1 in 10 people) are loss of appetite, vomiting, nausea (feeling sick), diarrhoea, lethargy (lack of energy) and pyrexia (fever). For the full list of all side effects reported with Procysbi, see the package leaflet.

Procysbi must not be used in people who are hypersensitive (allergic) to any form of mercaptamine or any of the other ingredients, or to penicillamine. It must also not be used in women who are breastfeeding.

Why is Procysbi approved?

The Agency’s Committee for Medicinal Products for Human Use (CHMP) decided that Procysbi’s benefits are greater than its risks and recommended that it be approved for use in the EU. The CHMP noted that Procysbi was shown to be at least as effective as Cystagon at maintaining the amount of cystine in white blood cells at acceptable levels. The Committee also considered that the gastroresistant formulation, due to its less frequent administration, is expected to increase compliance with treatment and the quality of life of patients with cystinosis. Regarding its safety, the CHMP considered that the safety profile of mercaptamine is well established and the safety of Procysbi is expected to be similar to that of the reference medicine.

What measures are being taken to ensure the safe and effective use of Procysbi?

A risk-management plan has been developed to ensure that Procysbi is used as safely as possible. Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Procysbi, including the appropriate precautions to be followed by healthcare professionals and patients.

In addition, the company that markets Procysbi will provide educational material to all doctors expected to prescribe the medicine, containing important safety information including the risk that the medicine may be harmful to the unborn child.

Other information about Procysbi

The European Commission granted a marketing authorisation valid throughout the European Union for Procysbi on 6 September 2013.

Name Language First published Last updated
Procysbi : EPAR - Summary for the public BG = bălgarski 2013-10-03  
Procysbi : EPAR - Summary for the public ES = español 2013-10-03  
Procysbi : EPAR - Summary for the public CS = čeština 2013-10-03  
Procysbi : EPAR - Summary for the public DA = dansk 2013-10-03  
Procysbi : EPAR - Summary for the public DE = Deutsch 2013-10-03  
Procysbi : EPAR - Summary for the public ET = eesti keel 2013-10-03  
Procysbi : EPAR - Summary for the public EL = elliniká 2013-10-03  
Procysbi : EPAR - Summary for the public EN = English 2013-10-03  
Procysbi : EPAR - Summary for the public FR = français 2013-10-03  
Procysbi : EPAR - Summary for the public IT = italiano 2013-10-03  
Procysbi : EPAR - Summary for the public LV = latviešu valoda 2013-10-03  
Procysbi : EPAR - Summary for the public LT = lietuvių kalba 2013-10-03  
Procysbi : EPAR - Summary for the public HU = magyar 2013-10-03  
Procysbi : EPAR - Summary for the public MT = Malti 2013-10-03  
Procysbi : EPAR - Summary for the public NL = Nederlands 2013-10-03  
Procysbi : EPAR - Summary for the public PL = polski 2013-10-03  
Procysbi : EPAR - Summary for the public PT = português 2013-10-03  
Procysbi : EPAR - Summary for the public RO = română 2013-10-03  
Procysbi : EPAR - Summary for the public SK = slovenčina 2013-10-03  
Procysbi : EPAR - Summary for the public SL = slovenščina 2013-10-03  
Procysbi : EPAR - Summary for the public FI = suomi 2013-10-03  
Procysbi : EPAR - Summary for the public SV = svenska 2013-10-03  
Procysbi : EPAR - Summary for the public HR = Hrvatski 2013-10-03  

This EPAR was last updated on 27/01/2016 .

Authorisation details

Product details

Product details for Procysbi
NameProcysbi
Agency product numberEMEA/H/C/002465
Active substance

mercaptamine bitartrate

International non-proprietary name (INN) or common name

mercaptamine

Therapeutic area Cystinosis
Anatomical therapeutic chemical (ATC) code A16AA04
Treatment of rare diseases

This medicine has an "orphan designation" which means that it is used to treat life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the European Union, or are medicines which, for economic reasons, would be unlikely to be developed without incentives.

Publication details

Publication details for Procysbi
Marketing-authorisation holder

Raptor Pharmaceuticals Europe BV

Revision4
Date of issue of marketing authorisation valid throughout the European Union06/09/2013

Contact address:

Raptor Pharmaceuticals Europe BV
Naritaweg 165
Telestone 8 
1043 BW Amsterdam 
The Netherlands

Product information

Product information

05/01/2016  Procysbi -EMEA/H/C/002465 -IB/0011

Name Language First published Last updated
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27
Procysbi : EPAR - Product Information HR = Hrvatski 2013-10-03 2016-01-27

Contents

  • Annex I - Summary of product characteristics
  • Annex IIA - Manufacturing-authorisation holder responsible for batch release
  • Annex IIB - Conditions of the marketing authorisation
  • Annex IIIA - Labelling
  • Annex IIIB - Package leaflet

Please note that the size of the above document can exceed 50 pages.

You are therefore advised to be selective about which sections or pages you wish to print.


Name Language First published Last updated
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  
Procysbi : EPAR - All Authorised presentations HR = Hrvatski 2013-10-03  

Pharmacotherapeutic group

Other alimentary-tract and metabolism products

Therapeutic indication

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.

Assessment History

Changes since initial authorisation of medicine

Name Language First published Last updated
Procysbi : EPAR - Procedural steps taken and scientific information after authorisation HR = Hrvatski 2014-09-04 2016-01-27

Initial marketing-authorisation documents

Name Language First published Last updated
Procysbi : EPAR - Public assessment report HR = Hrvatski 2013-10-03  
CHMP summary of positive opinion for Procysbi HR = Hrvatski 2013-06-28  

Authorised

This medicine is approved for use in the European Union

Related information

More information on Procysbi