EU/3/14/1301: Orphan designation for the treatment of idiopathic pulmonary fibrosis

Idiopathic pulmonary fibrosis is a long-term disease of the lungs characterised by the progressive deposition of collagen and fibrous tissue in the lungs. This causes the lung tissue to become thick and to form scars. As a result, 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 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 not more than 3 in 10,000 people in the European Union (EU). This was equivalent to a total of not more than 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 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 511,100,000 (Eurostat 2014).

At the time of designation, Esbriet (pirfenidone) was the only medicine authorised in the EU to treat mild to moderate idiopathic pulmonary fibrosis.

The sponsor has provided sufficient information to show that humanised anti-alpha ν beta 6 monoclonal antibody might be of significant benefit to patients with idiopathic pulmonary fibrosis based on results from early studies showing a reduction in fibrosis and because the medicine works in a different way to pirfenidone, specifically targeting 'alpha ν beta 6', a protein involved in the formation of fibrous tissue. These assumptions will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

This medicine is a monoclonal antibody, a type of protein that has been designed to recognise and attach to a specific target. The target for this medicine is a protein called alpha ν beta 6, which is found in high amounts in the lungs of patients with idiopathic pulmonary fibrosis, where it plays a role in the formation of fibrous tissue.

By attaching to alpha ν beta 6 and blocking its actions, the medicine is expected to reduce the formation of fibrous tissue in the lungs and thereby improve the patient's symptoms.

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. Orphan designation had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 12 June 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.