EU/3/14/1262 - orphan designation for prevention of bronchopulmonary dysplasia
recombinant human surfactant protein D
OrphanHuman
Overview
On 11 April 2014, orphan designation (EU/3/14/1262) was granted by the European Commission to Dr Ulrich Granzer, Germany, for recombinant human surfactant protein D for the prevention of bronchopulmonary dysplasia.
The sponsorship was transferred to Granzer Regulatory Consulting & Services GmbH, Germany in March 2022.
Bronchopulmonary dysplasia is a lung disease affecting premature babies who have been on prolonged mechanical ventilation (using a machine that supplies oxygen to help with breathing). In premature babies the lungs are immature and do not have enough surfactant, a substance lining the lungs and airways that allows them to expand properly. Bronchopulmonary dysplasia is caused by the constant high pressure of the oxygen supplied through mechanical ventilation, which causes inflammation and injury to the lungs and blocks the normal development of the alveoli (air sacs in the lungs). This leads to difficulty breathing and weight loss.
Bronchopulmonary dysplasia is a long-term and life-threatening condition because the 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 between 51,000 and 153,000 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.
*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 511,100,000 (Eurostat 2014).
At the time of designation, no satisfactory methods of prevention were authorised in the EU for bronchopulmonary dysplasia.
Recombinant human surfactant protein D is one of the components of the natural surfactant present in the lungs. The medicine is expected to work by replacing the missing surfactant protein D in the lungs of premature infants who are at risk of developing bronchopulmonary dysplasia. This should improve the child's breathing function by reducing inflammation to the lungs and allowing sufficient oxygen to be taken up.
At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.
At the time of submission, no clinical trials with the medicine in patients with bronchopulmonary dysplasia had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for bronchopulmonary dysplasia 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 6 February 2014 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 surfactant protein D
- Intended use
- Prevention of bronchopulmonary dysplasia
- Orphan designation status
- Positive
- EU designation number
- EU/3/14/1262
- Date of designation
- Sponsor
Granzer Regulatory Consulting & Services GmbH
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: