EU/3/16/1692: 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 long-term debilitating and life-threatening disease because the lungs gradually lose their ability to work properly.

At the time of designation, idiopathic pulmonary fibrosis affected approximately 3.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 180,000 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 513,700,000 (Eurostat 2016).

At the time of designation, Esbriet (pirfenidone) and Ofev (nintedanib) were authorised in the EU to treat 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 the laboratory show that it reduces pulmonary hypertension (high blood pressure in the blood vessels of the lungs, which is a common complication of the condition) as well as lung fibrosis (scarring). This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

This medicine stimulates a receptor called the angiotensin II type 2 receptor (AT2R). This receptor is found at high levels in damaged tissue, where it is thought to be involved in protecting tissues. Exactly how the medicine works in the condition is not clear, but it is believed that by attaching to AT2R, it reduces the production of inflammatory substances and the development of hard, fibrous tissue in the lungs, including in the blood vessels, thus controlling symptoms of the disease.

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with this medicine in patients with idiopathic pulmonary fibrosis had been started.

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 16 June 2016 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.