EU/3/16/1620 - orphan designation for treatment of retinitis pigmentosa
Allogeneic fetal human retinal progenitor cells expanded ex vivo
OrphanHuman
Overview
On 17 February 2016, orphan designation (EU/3/16/1620) was granted by the European Commission to Voisin Consulting S.A.R.L., France, for allogeneic fetal human retinal progenitor cells expanded ex vivo for the treatment of retinitis pigmentosa.
In October 2021, Voisin Consulting S.A.R.L. changed name to Voisin Consulting Life Sciences.
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.
At the time of designation, retinitis pigmentosa affected less than 3.7 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 190,000 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 513,700,000 (Eurostat 2016).
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.
The medicine contains retinal progenitor cells (or precursor cells) obtained from fetal tissue and grown in the laboratory.
The medicine is intended for injection into the vitreous humour (the jelly-like fluid) of the eye. Once present in the eye, the progenitor cells are expected to produce proteins called neurotrophic factors which may encourage the growth of the patient's own retinal cells and help protect retinal cells from further damage. This is expected to slow the progress of the disease and improve the patient's ability to see.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with this medicine in patients with retinitis pigmentosa were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for retinitis pigmentosa. Orphan designation of this medicine had been granted in the United States for the condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 21 January 2016 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
- Allogeneic fetal human retinal progenitor cells expanded ex vivo
- Intended use
- Treatment of retinitis pigmentosa
- Orphan designation status
- Positive
- EU designation number
- EU/3/16/1620
- Date of designation
- Sponsor
Voisin Consulting Life Sciences
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:
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: