EU/3/13/1135 - orphan designation for treatment of retinitis pigmentosa
recombinant human nerve growth factor
OrphanHuman
Overview
On 7 June 2013, orphan designation (EU/3/13/1135) was granted by the European Commission to Dompé S.p.A., Italy, for recombinant human nerve growth factor for the treatment of retinitis pigmentosa.
The sponsorship was transferred to Dompé farmaceutici s.p.a., Italy, in March 2015.
For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.
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 approximately 3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 154,000 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. At the time of designation, this represented a population of 512,200,000 (Eurostat 2013).
Human nerve growth factor (NGF) is a protein produced in the body that plays an important role in the growth and survival of nerve cells, including the cells of the retina. Patients with retinitis pigmentosa have lower than normal levels of NGF in the retina. Recombinant human NGF given as eye drops to patients with retinitis pigmentosa is expected to replace the missing NGF and improve the survival of retina cells, thereby slowing the development of the disease and helping to preserve vision.
The medicine is produced by a method known as 'recombinant DNA technology': it is made by bacteria into which a gene (DNA) has been introduced that makes them able to produce NGF.
At the time of submission of the application for orphan designation, the evaluation of the effects of recombinant human nerve growth factor in experimental models was ongoing, but no clinical trials with the medicine in patients with retinitis pigmentosa had been started.
At the time of submission, the medicine 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 17 April 2013 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
- recombinant human nerve growth factor
- Intended use
- Treatment of retinitis pigmentosa
- Orphan designation status
- Positive
- EU designation number
- EU/3/13/1135
- Date of designation
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: