EU/3/14/1341 - orphan designation for treatment of cystinosis
Cysteamine hydrochloride
OrphanHuman
Overview
On 15 October 2014, orphan designation (EU/3/14/1341) was granted by the European Commission to Lucane Pharma SA, France, for cysteamine hydrochloride for the treatment of cystinosis.
Cystinosis is an inherited disease in which the amino acid cystine builds up within cells. The cystine forms crystals that can damage the organs, especially the kidneys and the eyes. Cystinosis is caused by abnormalities in a protein called cystinosin, which normally helps to remove excess cystine from cells.
Cystinosis is a long-term debilitating condition that can lead to sight loss and intolerance to light and which may be life threatening because it can lead to kidney failure if left untreated.
At the time of designation, cystinosis affected approximately 0.15 in 10,000 people in the European Union (EU). This was equivalent to a total of around 7,600 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).
*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 511,100,000 (Eurostat 2014).
At the time of application for orphan designation, two medicines (Cystagon and Procysbi) containing cysteamine were authorised in the EU for use by mouth in the treatment of cystinosis. Ophthalmic (eye) formulations of cysteamine were not authorised in the EU, but were made up locally and used for the management of eye symptoms.
The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with cystinosis because the existing authorised medicines containing cysteamine given by mouth cannot adequately treat deposits of cystine in the eyes, whereas results from published studies suggest that this medicine, which is formulated as eye drops, can reduce such deposits.This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Cysteamine hydrochloride works by reacting with cystine to form other substances that are able to be removed from the cells. As a result, the amount of cystine in the cells is reduced, limiting the amount of organ damage.
The sponsor has provided data in experimental models from the published literature to support its application for orphan designation.
At the time of submission of the application for orphan designation, no clinical trials with this medicine in patients with cystinosis had been started by the sponsor.
At the time of submission, the medicine was not authorised anywhere in the EU for cystinosis or designated as an orphan medicinal product elsewhere for this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 4 September 2014 recommending the granting of this designation.
- the seriousness of the condition;
- the existence of alternative methods of diagnosis, prevention or treatment;
- either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.
Key facts
- Active substance
- Cysteamine hydrochloride
- Medicine name
- Dropcys
- Intended use
- Treatment of cystinosis
- Orphan designation status
- Positive
- EU designation number
- EU/3/14/1341
- Date of designation
- Sponsor
Lucane Pharma SA
172 rue de Charonne
75011 Paris
France
Tel. +33 1 53 86 87 50
Fax +33 1 43 71 14 96
E-mail: info@lucanepharma.com
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: