EU/3/16/1681

On 27 June 2016, orphan designation (EU/3/16/1681) was granted by the European Commission to Dr Alain Vicari, France, for humanised monoclonal antibody targeting interleukin-15 for the treatment of eosinophilic oesophagitis.

What is eosinophilic oesophagitis?

Eosinophilic oesophagitis is a disease characterised by inflammation of the oesophagus (the tube that leads from the mouth to the stomach) caused by excess of a type of white blood cell called eosinophils. The main symptoms of the disease are dysphagia (difficulty swallowing), which may lead to food getting stuck in the oesophagus, heartburn and chest pain.

Eosinophilic oesophagitis is a long-term debilitating disease that leads to oesophageal stenosis (narrowing of the oesophagus), and scarring and fragility of the oesophageal wall.

What is the estimated number of patients affected by the condition?

At the time of designation, eosinophilic oesophagitis affected approximately 3.8 in 10,000 people in the European Union (EU). This was equivalent to a total of around 195,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

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* 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).

What treatments are available?

At the time of designation, no satisfactory methods were authorised in the EU for the treatment of eosinophilic oesophagitis. As food allergy was thought to be a possible cause of the disease, certain food was excluded from the diet. Corticosteroids were given to reduce inflammation and improve symptoms. Oesophageal dilation (widening) was performed in some patients but it carries the risk of complication such as perforation of the oesophagus.

How is this medicine expected to work?

This medicine is a monoclonal antibody (a type of protein) that has been designed to attach to interleukin 15 (IL-15). IL-15 is produced at high levels in the oesophagus of patients with eosinophilic oesophagitis and is involved in attracting eosinophils and inducing inflammation. By attaching to IL-15, the medicine blocks its activity, reducing the number of eosinophils in the oesophagus and relieving inflammation and the symptoms of the disease.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with eosinophilic oesophagitis had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for eosinophilic oesophagitis 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 19 May 2016 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

• 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.