EU/3/13/1205 - orphan designation for treatment of eosinophilic oesophagitis
Human monoclonal antibody against human interleukin 13
OrphanHuman
Overview
On 13 November 2013, orphan designation (EU/3/13/1205) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for human monoclonal antibody against human interleukin 13 for the treatment of eosinophilic oesophagitis.
The sponsorship was transferred to Novartis Europharm Limited, Ireland, in May 2018.
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 acid regurgitation (acid flowing up into the mouth).
Eosinophilic oesophagitis is a long-term debilitating disease that leads to oesophageal stenosis (narrowing of the oesophagus), which can only be treated with invasive procedures.
At the time of designation, eosinophilic oesophagitis affected less than 5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 256,000 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 512,200,000 (Eurostat 2013).
At the time of designation, no satisfactory methods were authorised in the EU for the treatment of eosinophilic oesophagitis. As allergy was thought to be a possible cause of the disease, allergens were excluded from the diet. Oesophageal dilation (widening) was performed in some patients, although it carries the risk of complication such as perforation of the oesophagus.
This medicine is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to interleukin 13 (IL-13). IL-13 is produced at high levels by immune cells in patients with eosinophilic oesophagitis and is involved in attracting eosinophils and inducing inflammation. By attaching to IL-13, the medicine blocks its activity, reducing the number of eosinophils in the oesophagus and relieving inflammation and the symptoms of the disease.
The effects of this medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, one clinical trial with this medicine in patients with eosinophilic oesophagitis had been completed and further studies were planned.
At the time of submission, this medicine was not authorised anywhere in the EU for eosinophilic oesophagitis. Orphan designation of the medicine has been granted in the United States for eosinophilic oesophagitis.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 9 October 2013 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.
Key facts
- Active substance
- Human monoclonal antibody against human interleukin 13
- Intended use
- Treatment of eosinophilic oesophagitis
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/13/1205
- Date of designation
- Sponsor
Novartis Europharm Limited
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Update history
| Date | Update |
|---|---|
| August 2022 | Please note that this product was withdrawn from the Union Register of orphan medicinal products in August 2022 on request of the Sponsor. |
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: