EU/3/20/2308

Bronchopulmonary dysplasia is a lung disease affecting premature babies who have been on prolonged mechanical ventilation (to artificially push 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 and 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 of the damage to the lungs, leading to lack of oxygen in the blood.

At the time of designation, the number of patients at risk of bronchopulmonary dysplasia was estimated to be approximately 2.6 people in 10,000 in the European Union (EU). This was equivalent to a total of around 135,000 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).

*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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).

At the time of designation, no satisfactory methods were authorised in the EU for the prevention of bronchopulmonary dysplasia.

The medicine contains cells called mesenchymal stromal cells (MSCs) obtained from donor umbilical cord and grown in a laboratory to increase their numbers. MSCs are thought to increase production of substances that prevent inflammation and oxygen-induced damage, protect lung cells and encourage the repair and development of lung tissue. This is expected to prevent the symptoms of bronchopulmonary dysplasia.

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with bronchopulmonary dysplasia had been started.

At the time of submission, allogeneic umbilical cord tissue-derived mesenchymal stromal cells ex vivo expanded were not authorised anywhere in the EU for the prevention of bronchopulmonary dysplasia or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 16 July 2020, 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.