EU/3/20/2309: Orphan designation for the treatment of idiopathic pulmonary fibrosis

Idiopathic pulmonary fibrosis is a disease in which scar tissue (collagen and fibrous tissue) gradually builds up in the lungs, making their structure thicker and less flexible. As a result, the lungs no longer work normally, reducing the amount of oxygen that gets 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 3.2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 166,000 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).

*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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).

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 the medicine might be of significant benefit for patients with idiopathic pulmonary fibrosis. Early data suggest that the medicine may be more effective than authorised medicines in patients with IPF.

This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

The medicine blocks the action of a protein called galectin-3, which is involved in the production of fibrous tissue in organs including the lungs, and is thought to play a role in the development of IPF. By blocking the activity of galectin-3, the medicine is expected to reduce build-up of fibrous tissue in the lungs, thereby improving their functioning.

The effects of bis-(3-deoxy-3-(4-(3-fluorophenyl)-1H-1,2,3-triazol-1-yl)-beta-D-galactopyranosyl) sulfane 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 the treatment of idiopathic pulmonary fibrosis or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 16 July 2020, 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.