EU/3/04/210 - orphan designation for treatment of bronchiolitis obliterans syndrome
ciclosporin
OrphanHuman
Overview
On 24 May 2017, orphan designation (EU/3/04/210) was granted by the European Commission to PARI Pharma GmbH, Germany, for ciclosporin for the treatment of bronchiolitis obliterans syndrome.
The sponsorship was transferred to Breath Therapeutics GmbH, Germany, in August 2017.
The sponsor’s address was updated in July 2019.
The sponsorship was transferred to Zambon S.p.A. in March 2021.
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 52,000 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) specifically for the treatment of bronchiolitis obliterans syndrome. Patients received immunosuppressant medicines that reduce the activity of immune cells, with the aim of reducing the immune system’s ability to attack the patient’s lungs.
The medicine contains ciclosporin, a medicine that has been used in the EU since the 1980s to suppress the immune system after a transplant. It is to be inhaled into the lungs as a mist. The ciclosporin is contained in tiny fat particles called liposomes that help it to be absorbed directly into the affected tissues, where it can reduce the activity of the immune cells attacking the lungs without much effect on the rest of the body. This is expected to reduce the development of scarring and obstruction in the airways, and so improve survival.
The effects of ciclosporin for inhalation use were evaluated in experimental models. At the time of submission of the application for orphan designation, no clinical trials in patients with graft rejection after lung transplantation were initiated.
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, this medicine was not authorised anywhere in the EU for treatment of bronchiolitis obliterans syndrome.
The medicine had been designated orphan on 29 July 2004 for the treatment of graft rejection after lung transplantation. At the request of the sponsor and having assessed the additional data submitted, the COMP adopted an opinion on 11 May 2017 recommending the change of the orphan condition from graft rejection after lung transplantation to bronchiolitis obliterans syndrome.
Orphan designation of the medicine has been granted in the United States for the treatment of bronchiolitis obliterans.
- 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
- ciclosporin
- Intended use
- Treatment of bronchiolitis obliterans syndrome
- Orphan designation status
- Positive
- EU designation number
- EU/3/04/210
- Date of designation
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: