EU/3/11/903

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Orphan designation

On 27 September 2011, orphan designation (EU/3/11/903) was granted by the European Commission to ActoGeniX N.V., Belgium, for genetically modified Lactococcus lactis bacteria containing the human trefoil factor 1 gene for the prevention of oral mucositis in head and neck cancer patients undergoing radiation therapy.

What is oral mucositis?

Oral mucositis is inflammation of the lining of the mouth which results in symptoms such as ulcers, severe pain, bleeding and inflammation. Patients with head and neck cancer are prone to oral mucositis because of the radiation therapy they receive. Radiation therapy employs high-dose X-rays or other high-energy rays to kill the cancer cells in the head and neck. Unfortunately the radiation therapy is also likely to cause inflammation in the surrounding cells and tissues.

Oral mucositis in head and neck cancer patients undergoing radiation therapy is a long-term debilitating disease because it can lead to pain, poor nutrition, infections and it makes the patient less likely to comply with their radiotherapy treatment.

What is the estimated number of patients at risk of developing the condition?

At the time of designation, the number of patients in the European Union (EU) with head and neck cancer at risk of oral mucositis following radiation therapy was estimated to be less than 4 in 10,000 people *. This is equivalent to a total of fewer than 203,000 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 27), Norway, Iceland and Liechtenstein. This represents a population of 506,300,000 (Eurostat 2011).

What methods of prevention are available?

At the time of designation, mouth rinses containing calcium phosphate and povidone iodine were authorised in some EU countries for preventing oral mucositis in head and neck cancer patients undergoing radiation therapy.
The sponsor has provided sufficient information to show that genetically modified Lactococcus lactis bacteria containing the human trefoil factor 1 gene might be of significant benefit for mucositis in head and neck cancer patients undergoing radiotherapy because it works in a different way to existing methods and early studies show that it might offer benefits to patients with this condition, possibly in combination with existing methods. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan statusAt the time of designation, mouth rinses containing calcium phosphate and povidone iodine were authorised in some EU countries for preventing oral mucositis in head and neck cancer patients undergoing radiation therapy.

The sponsor has provided sufficient information to show that genetically modified Lactococcus lactis bacteria containing the human trefoil factor 1 gene might be of significant benefit for mucositis in head and neck cancer patients undergoing radiotherapy because it works in a different way to existing methods and early studies show that it might offer benefits to patients with this condition, possibly in combination with existing methods. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

The medicine is a ‘gene therapy product’ that works by delivering specific genes into the body with the purpose of providing a specific protein. The medicine contains bacteria called Lactococcus lactis that have been genetically modified so that they produce a protein called human trefoil factor 1. This is a natural human protein found in the lining of the mouth and the gut where it is involved in protecting and repairing the cells of this lining. The medicine is expected to be available as a mouth rinse. When patients use the mouth rinse, the bacteria are expected to enter the patient’s mouth and produce the human trefoil factor 1, thus helping to protect and repair the cells of the mouth lining and to prevent oral mucositis.

What is the stage of development of this medicine?

The effects of genetically modified Lactococcus lactis bacteria containing the human trefoil factor 1 gene have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicinal product in head and neck cancer patients undergoing radiation therapy were ongoing.

At the time of submission, the medicinal product was not authorised anywhere in the EU for the prevention of oral mucositis in head and neck cancer patients undergoing radiation therapy 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 8 July 2011 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.

Key facts

Product details for <p>Genetically modified Lactococcus lactis bacteria containing the <em>human trefoil factor 1</em> gene</p>
Active substanceGenetically modified Lactococcus lactis bacteria containing the human trefoil factor 1 gene
Medicine Name
Disease/conditionPrevention of oral mucositis in head and neck cancer patients undergoing radiation therapy
Date of decision27/09/2011
OutcomePositive
Orphan decision numberEU/3/11/903

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor’s contact details

ActoGeniX N.V.
Technologiepark 4
B-9052 Zwijnaarde
Belgium
Tel. +32 9261 0600
Fax +32 9261 0619
E-mail: info@actogenix.com

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases which includes a directory of patients’ organisations registered in Europe.
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.