EU/3/17/1907 - orphan designation for prevention of bronchopulmonary dysplasia

recombinant fragment of human surfactant protein-D

Overview

On 23 August 2017, orphan designation (EU/3/17/1907) was granted by the European Commission to Trimunocor Ltd, United Kingdom, for recombinant fragment of human surfactant protein-D for the prevention of bronchopulmonary dysplasia.

The sponsorship was transferred to Premier Research Group S.L., Spain, in August 2019.

What is bronchopulmonary dysplasia?

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.

What is the estimated number of patients at risk of developing the condition?

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 103,000 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).

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* 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 515,700,000 (Eurostat 2017).

What methods of prevention are available?

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

How is this medicine expected to work?

This medicine consists of a fragment of surfactant protein-D, a protein found in the lungs that helps reduce inflammation. By giving the medicine in the first few days of life, it is expected to overcome the deficiency of surfactant protein-D in premature babies and so prevent the inflammatory process that can cause bronchopulmonary dysplasia.

What is the stage of development of this medicine?

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 at risk of developing 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 13 July 2017 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria

• 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.

EU/3/17/1907: Public summary of opinion on orphan designation: Recombinant fragment of human surfactant protein-D for the prevention of bronchopulmonary dysplasia

Adopted Reference Number: EMA/507449/2017

Key facts

• Active substance:
  recombinant fragment of human surfactant protein-D
• Medicine name:
  -
• Intended use:
  Prevention of bronchopulmonary dysplasia
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/17/1907
• Date of designation:
  23/08/2017
• Sponsor:

  Premier Research Group S.L.; Camino De La Zarzuela 19; 28023 Madrid; Spain; Tel. [phone]; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.