EU/3/15/1503 - orphan designation for prevention of bronchopulmonary dysplasia
allogeneic ex-vivo-expanded human umbilical cord blood-derived mesenchymal stem cells
OrphanHuman
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Overview
On 19 June 2015, orphan designation (EU/3/15/1503) was granted by the European Commission to PSR Group B.V., the Netherlands, for allogeneic ex-vivo-expanded human umbilical cord blood-derived mesenchymal stem cells for the prevention of bronchopulmonary dysplasia.
Bronchopulmonary dysplasia is a lung disease affecting premature babies who have been on prolonged mechanical ventilation (to artificially drive oxygen-rich air into the lungs). The constant high pressure of the oxygen from mechanical ventilation can cause inflammation and damage to the cells in the lungs, as well as hinder the normal development of the lungs. This can lead to long-term breathing problems and weight loss.
Bronchopulmonary dysplasia is a long-term and life-threatening condition because the damaged and underdeveloped lungs may not work properly.
At the time of designation, the number of patients at risk of bronchopulmonary dysplasia was estimated to be between 1 and 3 people in 10,000 in the European Union (EU). This was equivalent to a total of between 51,000 and 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 at risk of developing 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 512,900,000 (Eurostat 2015).
At the time of designation, no satisfactory methods were authorised in the EU for the prevention of bronchopulmonary dysplasia.
This medicine is made of stem cells extracted from umbilical cord blood donated after birth and then grown in a laboratory, in order to increase their numbers. Although it is not fully understood how they work, when these cells are given to premature babies at risk of bronchopulmonary dysplasia, they could potentially mature and replace the damaged cells in the tissue of the lungs. The medicine is expected to reduce the activity of the immune cells that cause inflammation and damage to the lungs.
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 the medicine in patients at high risk of bronchopulmonary dysplasia were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for bronchopulmonary dysplasia. Orphan designation of the medicine had been granted in the United States for the prevention of this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 13 May 2015 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 ex-vivo-expanded human umbilical cord blood-derived mesenchymal stem cells
- Medicine name
- -
- Intended use
- Prevention of bronchopulmonary dysplasia
- Orphan designation status
- Positive
- EU designation number
- EU/3/15/1503
- Date of designation
- Sponsor
Ergomed B.V.
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Update history
| Date | Update |
|---|---|
| November 2024 | The sponsor's name changed to Ergomed B.V. |
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: