EU/3/11/934: Orphan designation for the treatment of idiopathic pulmonary fibrosis

Idiopathic pulmonary fibrosis is a long-term disease of the lungs characterised by the progressive formation of hard tissue in the lining of the lungs. 'Idiopathic' means that the cause of the disease is unknown. As the tissue becomes thicker and forms scars, the lungs become unable to work normally, reducing the transfer of oxygen from the air into the blood. Patients with idiopathic pulmonary fibrosis have a persistent cough, frequent lung infections and severe shortness of breath that worsens over time.

Idiopathic pulmonary fibrosis is a life-threatening and long-term debilitating disease because the lungs gradually lose their ability to work properly.

At the time of designation, idiopathic pulmonary fibrosis affected approximately 1.5 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 76,000 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 27), Norway, Iceland and Liechtenstein. This represents a population of 506,300,000 (Eurostat 2011).

At the time of designation, medicines such as corticosteroids, cyclophosphamide, cyclosporin and azathioprine were used in the EU to treat idiopathic pulmonary fibrosis. Pirfenidone (Esbriet) was authorised in the EU for the treatment of mild to moderate idiopathic pulmonary fibrosis.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with idiopathic pulmonary fibrosis because early studies in experimental models show that it works in a different way to existing treatments and might improve the outcome of patients with this condition. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

The active substance in the medicine is a 'c-Jun amino-terminal kinase (JNK) inhibitor'. This means that it blocks the action of an enzyme called JNK, which is involved in several processes within the cells, including inflammation and the formation of scar tissue. By blocking the actions of this enzyme, the medicine is expected to improve the lung condition of patients with idiopathic pulmonary fibrosis.

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 idiopathic pulmonary fibrosis were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for idiopathic pulmonary fibrosis 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 7 October 2011 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.