Cystadrops

mercaptamine

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This is a summary of the European public assessment report (EPAR) for Cystadrops. It explains how the 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 Cystadrops.

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

What is Cystadrops and what is it used for?

Cystadrops is an eye medicine used in patients from 2 years of age who have cystinosis. Cystinosis is an inherited disease where a natural substance called cystine builds up in the body forming damaging crystals, particularly in the kidneys and cornea (the transparent layer in front of the eye). Cystadrops is used to reduce the build-up of cystine crystals in the cornea.

Because the number of patients with cystinosis is low, the disease is considered ‘rare’, and Cystadrops was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 7 November 2008.

Cystadrops contains the active substance mercaptamine (also known as cysteamine).

How is Cystadrops used?

Cystadrops can only be obtained with a prescription and treatment should be started under the supervision of a doctor experienced in the management of cystinosis.

Cystadrops is available as eye drops. The recommended dose is 1 drop in each eye 4 times a day during waking hours. The doctor may decrease the dose progressively to 1 drop a day depending on the results of eye examinations. Treatment should be continued long-term.

For further information, see the package leaflet.

How does Cystadrops work?

Damage from the build-up of cystine crystals inside the cells of the cornea can cause serious vision problems. The active substance in Cystadrops, mercaptamine, reacts with cystine to dissolve it and to form substances that can be removed from the cells. When it is applied to the eye, the amount of cystine in the cells of the cornea decreases, limiting damage to the eye.

What benefits of Cystadrops have been shown in studies?

Cystadrops has been compared with another less concentrated mercaptamine eye drop solution in one main study involving 32 patients from 2 years of age with cystinosis. The main measure of effectiveness was based on the reduction in corneal cystine crystals as seen under the microscope and quantified using a score called the IVCM score. The score ranges from 0 to 28, with 0 corresponding to no crystals. At the start of the study patients in both groups had an average score of 10.

Cystadrops was shown to be more effective than the comparator at decreasing corneal cystine crystals after 3 months of treatment: in patients using Cystadrops the IVCM score decreased by 4.6 points compared with a decrease of 0.5 points in patients using the comparator. Treatment with Cystadrops also led to reductions in photophobia (eye discomfort in bright light).

What are the risks associated with Cystadrops?

The most common side effects with Cystadrops (which may affect more than 1 in 10 people) are related to the eye and include pain, itching and irritation in the eye, increased lacrimation (watering eyes), blurred vision and ocular hyperaemia (red eye). These effects are usually mild or moderate and do not last.

For the full list of all side effects and restrictions with Cystadrops, see the package leaflet.

Why is Cystadrops approved?

Mercaptamine eye drop solutions made up locally in pharmacies or hospitals have been used for many years for the management of eye symptoms of cystinosis. In addition, Cystadrops has been shown in a study to be effective at decreasing corneal cystine crystals. The medicine also improved other symptoms of cystinosis such as photophobia. Regarding safety, although side effects related to the eye are very common, they are usually manageable.

The Agency’s Committee for Medicinal Products for Human Use (CHMP) decided that Cystadrops’s benefits are greater than its risks and recommended that it be approved for use in the EU.

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

Recommendations and precautions to be followed by healthcare professionals and patients for the safe and effective use of Cystadrops have been included in the summary of product characteristics and the package leaflet.

Other information about Cystadrops

The European Commission granted a marketing authorisation valid throughout the European Union for Cystadrops on 19 January 2017.

For more information about treatment with Cystadrops, read the package leaflet (also part of the EPAR) or contact your doctor or pharmacist.

The summary of the opinion of the Committee for Orphan Medicinal Products for Cystadrops can be found on the Agency’s website.

Name Language First published Last updated
Cystadrops : EPAR - Summary for the public BG = bălgarski 2017-02-22  
Cystadrops : EPAR - Summary for the public ES = español 2017-02-22  
Cystadrops : EPAR - Summary for the public CS = čeština 2017-02-22  
Cystadrops : EPAR - Summary for the public DA = dansk 2017-02-22  
Cystadrops : EPAR - Summary for the public DE = Deutsch 2017-02-22  
Cystadrops : EPAR - Summary for the public ET = eesti keel 2017-02-22  
Cystadrops : EPAR - Summary for the public EL = elliniká 2017-02-22  
Cystadrops : EPAR - Summary for the public EN = English 2017-02-22  
Cystadrops : EPAR - Summary for the public FR = français 2017-02-22  
Cystadrops : EPAR - Summary for the public IT = italiano 2017-02-22  
Cystadrops : EPAR - Summary for the public LV = latviešu valoda 2017-02-22  
Cystadrops : EPAR - Summary for the public LT = lietuvių kalba 2017-02-22  
Cystadrops : EPAR - Summary for the public HU = magyar 2017-02-22  
Cystadrops : EPAR - Summary for the public MT = Malti 2017-02-22  
Cystadrops : EPAR - Summary for the public NL = Nederlands 2017-02-22  
Cystadrops : EPAR - Summary for the public PL = polski 2017-02-22  
Cystadrops : EPAR - Summary for the public PT = português 2017-02-22  
Cystadrops : EPAR - Summary for the public RO = română 2017-02-22  
Cystadrops : EPAR - Summary for the public SK = slovenčina 2017-02-22  
Cystadrops : EPAR - Summary for the public SL = slovenščina 2017-02-22  
Cystadrops : EPAR - Summary for the public FI = suomi 2017-02-22  
Cystadrops : EPAR - Summary for the public SV = svenska 2017-02-22  
Cystadrops : EPAR - Summary for the public HR = Hrvatski 2017-02-22  

This EPAR was last updated on 09/03/2017 .

Authorisation details

Product details

Product details for Cystadrops
NameCystadrops
Agency product numberEMEA/H/C/003769
Active substance

mercaptamine hydrochloride

International non-proprietary name (INN) or common name

mercaptamine

Therapeutic area Cystinosis
Anatomical therapeutic chemical (ATC) code S01XA21
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 Cystadrops
Marketing-authorisation holder

Orphan Europe S.A.R.L.

Revision1
Date of issue of marketing authorisation valid throughout the European Union19/01/2017

Contact address:

Orphan Europe S.A.R.L.
Immeuble "Le Wilson" 70
Avenue du Général de Gaulle
FR-92800 Puteaux
France

Product information

Product information

16/02/2017  Cystadrops -EMEA/H/C/003769 -IAIN/0001/G

Name Language First published Last updated
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09
Cystadrops : EPAR - Product Information HR = Hrvatski 2017-02-22 2017-03-09

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
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  
Cystadrops : EPAR - All Authorised presentations HR = Hrvatski 2017-02-22  

Pharmacotherapeutic group

Ophthalmologicals

Therapeutic indication

Cystadrops is indicated for the treatment of corneal cystine crystal deposits in adults and children from 2 years of age with cystinosis.

Assessment History

Changes since initial authorisation of medicine

Name Language First published Last updated
Cystadrops : EPAR - Procedural steps taken and scientific information after authorisation HR = Hrvatski 2017-03-09  

Initial marketing-authorisation documents

Name Language First published Last updated
Cystadrops : EPAR - Public assessment report HR = Hrvatski 2017-02-22  
CHMP summary of positive opinion for Cystadrops HR = Hrvatski 2016-10-14