EU/3/14/1279 - orphan designation for prevention of scarring post glaucoma filtration surgery
4-(4-Methoxy-phenylamino)-6-methylcarbamyl-quinoline-3-carboxylic acid
OrphanHuman
Overview
On 4 June 2014, orphan designation (EU/03/14/1279) was granted by the European Commission to Clanotech AB, Sweden, for 4-(4-methoxy-phenylamino)-6-methylcarbamyl-quinoline-3-carboxylic acid for the prevention of scarring post glaucoma filtration surgery.
Glaucoma is a disease in which the pressure in the eye rises because the fluid inside the eye cannot drain away properly. The pressure affects the optic nerve (the nerve that sends signals from the eye to the brain) and can cause loss of vision. Glaucoma can be treated using 'filtration surgery', when the surgeon makes a small hole in the sclera (the white of the eye), to create a drainage channel, so that the fluid that has built up in the eye can drain away. After surgery, as part of the body's normal healing process, specialised cells called fibroblasts build up scar tissue which helps to repair the wound. However, scarring can sometimes be excessive and seal the channel up, causing the pressure inside the eye to build up again.
Scarring post glaucoma filtration surgery is a long-term debilitating condition because it may lead to loss of vision.
At the time of designation, the number of patients at risk of scarring post glaucoma filtration surgery was estimated to be approximately 1.9 people in 10,000 in the European Union (EU). This was equivalent to a total of around 97,000 people*, and is below the ceiling for orphan designation which is 5 people in 10,000. This is based 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 designation, no satisfactory methods were authorised in the EU for the prevention of scarring post glaucoma filtration surgery. Medicines that block and kill fibroblasts, such as mitomycin C or 5-fluorouracil (which are also used to treat cancer), were applied to the eye during surgery to slow down the formation of scar tissue and prevent the channel from sealing up.
This medicine is expected to work by preventing the migration and adhesion of fibroblasts to the wound area. This is expected to help the wound in the eye to heal without excessive scarring, thus preventing pressure in the eye from building up again following glaucoma surgery.
At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.
At the time of submission, no clinical trials with the medicine in patients with scarring post glaucoma filtration surgery had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for scarring post glaucoma filtration surgery 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 9 April 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.
Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.
Key facts
- Active substance
- 4-(4-Methoxy-phenylamino)-6-methylcarbamyl-quinoline-3-carboxylic acid
- Intended use
- Prevention of scarring post glaucoma filtration surgery
- Orphan designation status
- Positive
- EU designation number
- EU/3/14/1279
- Date of designation
- Sponsor
Clanotech AB
Fogdevreten 2
SolnaS-171 65
Sweden
Tel. +46 703 747 179
E-mail: patrizia.caldirola@clanotech.se
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:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
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: