EU/3/20/2294: Orphan designation for the treatment of idiopathic pulmonary fibrosis

Idiopathic pulmonary fibrosis is a disease characterised by progressive deposition of collagen and fibrous tissue in the lungs. This causes the lung tissue to become thick and form scars. 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, 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 because early data suggested that sodium cromoglicate may reduce cough when given to patients already receiving either pirfenidone or nintedanib. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

*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).

Idiopathic pulmonary fibrosis is thought to be caused by excess activity of fibroblasts, a type of skin cell that produces collagen and other skin components. Sodium cromoglycate is expected to act on cells of the immune system (the body’s natural defences) called mast cells and prevent them from stimulating fibroblasts. When taken directly into the lungs using a nebulizer (a machine that changes a solution into an aerosol that the patient can breathe in), sodium cromoglicate is expected to prevent the build-up of fibrous tissue and improve symptoms of the disease. 

The effects of sodium cromoglicate have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with sodium cromoglicate in patients with idiopathic pulmonary fibrosis were ongoing.

At the time of submission, sodium cromoglicate was not authorised anywhere in the EU for the treatment of idiopathic pulmonary fibrosis. Orphan designation of sodium cromoglicate had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 20 May 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.