On 19 June 2013, orphan designation (EU/3/13/1140) was granted by the European Commission to ReNeuron Ltd, United Kingdom, for expanded human allogeneic neural retinal progenitor cells extracted from neural retina for the treatment of retinitis pigmentosa.
- What is retinitis pigmentosa?
Retinitis pigmentosa is a group of hereditary diseases of the eye that lead to progressive loss of sight. In patients with retinitis pigmentosa, cells in the retina (the light-sensitive surface at the back of the eye) become damaged and eventually die.
Retinitis pigmentosa is a long-term debilitating disease because it causes the patient’s sight to get worse, eventually leading to blindness.
- What is the estimated number of patients affected by the condition?
At the time of designation, retinitis pigmentosa affected approximately 3.7 in 10,000 people in the European Union (EU). This was equivalent to a total of around 188,000 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 27), Norway, Iceland and Liechtenstein. This represents a population of 509,000,000 (Eurostat 2013).
- What treatments are available?
At the time of designation, no satisfactory methods were authorised in the EU for treating retinitis pigmentosa. Patients with the condition were given sunglasses to slow down the damage to the retina, genetic counselling (discussion of the risks of passing the condition on to children) and general support.
- How is this medicine expected to work?
The medicine contains retinal progenitor cells (or precursor cells) obtained from the eye and grown in a laboratory.
The medicine is intended for injection into the eye, under the retina. Once inside the retina, the cells in the medicine are expected to develop into mature retinal cells replacing the damaged cells. This is expected to help improve the person’s sight.
- What is the stage of development of this medicine?
The effects of the medicinal product have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with the medicinal product in patients with retinitis pigmentosa had been started.
At the time of submission, the medicinal product was not authorised anywhere in the EU for retinitis pigmentosa 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 15 May 2013 recommending the granting of this designation.
- Opinions on orphan medicinal product designations are based on the following three criteria:
- 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.
|Name||Language||First published||Last updated|
|EU/3/13/1140: Public summary of opinion on orphan designation: Expanded human allogeneic neural retinal progenitor cells extracted from neural retina for the treatment of retinitis pigmentosa||(English only)||2013-07-11|
|Active substance||expanded human allogeneic neural retinal progenitor cells extracted from neural retina|
|Disease/condition||Treatment of retinitis pigmentosa|
|Date of decision||19/06/2013|
|Orphan decision number||EU/3/13/1140|
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Sponsor’s contact details
Pencoed Business Park
Bridgend CF35 5HY
Tel. +44 (0)20 3819 8400
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.