EU/3/10/756 - orphan designation for prevention of scarring post glaucoma filtration surgery
Tranilast
OrphanHuman
Overview
On 27 July 2010, orphan designation (EU/3/10/756) was granted by the European Commission to Altacor Ltd, United Kingdom, for tranilast for the prevention of scarring post glaucoma filtration surgery.
The sponsorship was transferred to Voisin Consulting S.A.R.L., France in November 2019.
In October 2021, Voisin Consulting S.A.R.L. changed name to Voisin Consulting Life Sciences.
Glaucoma is a disease in which the pressure in the eye rises because the fluid inside the eye cannot drain away. 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), so that the fluid that has built up in the eye can drain away. Scarring can occur after surgery if too much collagen (a type of protein) is laid down in the channel during healing. This can seal the channel up and cause the pressure inside the eye to build up again.
Scarring post glaucoma filtration surgery is a serious and 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 less than 3 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 152,000 people, and is below the threshold 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 27), Norway, Iceland and Liechtenstein. This represents a population of 506,500,000 (Eurostat 2010).
At the time of designation, no satisfactory methods were authorised in the EU for the prevention of scarring post glaucoma filtration surgery. Medicines that stop the growth of cells, such as 5-fluorouracil and mitomycin C (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 sealing up.
Tranilast is expected to work by blocking the release of 'transforming growth factor beta' (TGF-?), a protein involved in wound healing that can cause excessive collagen production and scarring. The reduction of TGF-? levels is expected to help the wound in the eye to heal without excessive scarring.
The sponsor of this application has not conducted any studies with tranilast. However, it has provided the results of studies in experimental models and of clinical trials from the published literature to support its application for orphan designation.
Tranilast is already marketed in Japan and South Korea for allergic conditions (including allergic conjunctivitis) and scarring of the skin.
At the time of submission, tranilast was not authorised anywhere in the EU for the prevention of 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 8 April 2010 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
- Tranilast
- Intended use
- Prevention of scarring post glaucoma filtration surgery
- Orphan designation status
- Positive
- EU designation number
- EU/3/10/756
- Date of designation
- Sponsor
Esperante Development B.V.
Update history
| Date | Update |
|---|---|
| January 2024 | The sponsorship was transferred from Voisin Consulting Life Sciences to Esperante Development B.V. |
| December 2023 | The sponsorship was transferred from Dlrc Pharma Services Limited to Voisin Consulting Life Sciences. |
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: