EU/3/20/2306: Orphan designation for the prevention of bronchopulmonary dysplasia

Bronchopulmonary dysplasia is a lung disease affecting premature babies who have been on prolonged mechanical ventilation (to 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 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 less than 2 people in 10,000 in the European Union (EU). This was equivalent to a total of fewer than 104,000 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.

Retinol, a form of vitamin A, plays an essential role in the growth and development of the lungs. Premature babies have low levels of retinol. Restoring normal levels of retinol is expected to improve lung development and help the lungs absorb oxygen better, thus reducing the need for mechanical ventilation and helping to prevent bronchopulmonary dysplasia. The medicine contains retinol in a form that can be given by injection, as very premature babies cannot absorb vitamin A properly if it is given by mouth.

Medicines containing vitamin A have been available in the EU for many years to treat deficiency of the vitamin.

The effects of retinol palmitate have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with retinol palmitate in patients with bronchopulmonary dysplasia had finished.

At the time of submission, medicines containing retinol palmitate were authorised in the EU for the treatment of vitamin A deficiency. However, retinol palmitate was 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 18 June 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.