EU/3/17/1842 - orphan designation for treatment of bronchiolitis obliterans syndrome
Recombinant human club cell 10 KDa protein
OrphanHuman
Overview
On 27 February 2017, orphan designation (EU/3/17/1842) was granted by the European Commission to EUDRAC Limited, United Kingdom, for recombinant human club cell 10 KDa protein for the treatment of bronchiolitis obliterans syndrome.
The sponsorship was transferred to EUDRAC GmbH, Germany, in April 2019.
Bronchiolitis obliterans syndrome is the most common complication affecting patients who have had a lung transplant. Rarely, it may also occur in patients who receive a bone marrow transplant. Bronchiolitis obliterans syndrome occurs when cells of the immune system (the body's natural defences) recognise the patient's lungs as 'foreign' and attack them. This leads to extensive scarring (fibrosis) that obstructs and damages the lungs, leading eventually to death.
Bronchiolitis obliterans syndrome is a debilitating and life-threatening disease due to the progressive damage to the lungs.
At the time of designation, bronchiolitis obliterans syndrome affected less than 1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 51,000 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 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 515,700,000 (Eurostat 2017).
At the time of designation, no satisfactory methods were authorised in the European Union (EU) for the treatment of bronchiolitis obliterans syndrome. Patients received medicines such as ciclosporin and corticosteroids. Treatment aimed to reduce the activity of immune cells, thereby reducing their ability to attack the patient's lungs.
The medicine is made of a protein called 'club cell 10 KDa protein' (CC10) which is normally produced by cells in the lungs and which is lacking in patients with bronchiolitis obliterans syndrome. CC10 plays a crucial role in repairing lung tissue following injury by blocking several inflammatory substances. In particular, it reduces the number of neutrophils (a type of immune cell) in the lungs and blocks the production of cytokines (messenger molecules of the immune system) which are involved in the inflammatory process. By increasing CC10 levels, the medicine is expected to help repair lung tissue and reduce the symptoms of bronchiolitis obliterans syndrome.
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 with bronchiolitis obliterans syndrome were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for bronchiolitis obliterans syndrome or designated as an orphan medicinal product elsewhere for this condition. The medicine had been granted orphan designation in the EU for bronchopulmonary dysplasia (a lung disease affecting premature babies).
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 19 January 2017 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 club cell 10 KDa protein
- Intended use
- Treatment of bronchiolitis obliterans syndrome
- Orphan designation status
- Positive
- EU designation number
- EU/3/17/1842
- Date of designation
- Sponsor
EUDRAC GmbH
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product it 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: