EU/3/07/522

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Orphan designation

On 31 January 2008, orphan designation (EU/3/07/522) was granted by the European Commission to Celtic Bio-Pharma Services Ltd, United Kingdom, for (manganese, dichloro [(4aR, 13aR, 17aR, 21aR)-1, 2, 3, 4, 4a, 5, 6, 12, 13, 13a, 14, 15, 16, 17, 17a, 18, 19, 20, 21, 21a-eicosahydro-11, 7-nitrilo-7H-dibenzo[ b,h] [1,4,7,10] tetraazacycloheptadecine-κN5, κN13, κN18, κN21, κN22]-), for the prevention of oral mucositis in head and neck cancer patients undergoing radiation therapy.

What is oral mucositis?

Radiation therapy is often used as a treatment option in selected tumours. The technique employs high-dose x-rays or other high-energy rays to kill cancer cells. Radiation therapy will not only kill the tumour cells, but will unfortunately also cause some degree of injury (e.g., inflammation) to the surrounding cells and tissues. Radiation therapy applied to tumours located in the head and neck region usually damages the inner lining (mucosa) of the mouth. The damage results in inflammation, ulcers, severe pain, bleeding and dryness of the mouth. This complication of radiation therapy is called “oral mucositis”. The condition is chronically debilitating as it causes severe pain and leads to difficulty or inability to swallow, and consequently to malnutrition.

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

At the time of designation, the number of patients at risk of oral mucositis in head and neck cancer patients undergoing radiation therapy was estimated to be approximately 2.8 people in 10,000 in the European Union (EU)*. This is equivalent to a total of around 140,000 people, which 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 based on data from the European Union (EU 27), Norway, Iceland and Lichtenstein. This represents a population of 498,000,000 (Eurostat 2006). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

What methods of prevention are available?

No satisfactory methods exist that were authorised at the time of application.

How is this medicine expected to work?

The medicinal product mimics the action of a natural component of the cells, an enzyme called superoxide dismutase. Superoxide dismutase is thought to have a double effect, firstly it can reduce inflammation, and secondarily it can decrease the rate of programmed cell death (apoptosis). As mucositis involves inflammation, the positive effect of the product over this process, as well as the reduced rate of apoptosis, is expected to have a beneficial effect in the condition.

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 (manganese, dichloro [(4aR, 13aR, 17aR, 21aR)-1, 2, 3, 4, 4a, 5, 6, 12, 13, 13a, 14, 15, 16, 17, 17a, 18, 19, 20, 21, 21a-eicosahydro-11, 7-nitrilo-7H-dibenzo[ b,h] [1,4,7,10] tetraazacycloheptadecine-κN5, κN13, κN18, κN21, κN22]-) in experimental models was ongoing; clinical trials in head and neck cancer patients receiving radiotherapy had not started yet.

The medicinal product was not marketed anywhere worldwide as a medicinal product for prevention of oral mucositis in head and neck cancer patients undergoing radiation therapy or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 December 2007 recommending the granting of this designation.

Opinions on orphan medicinal products designations are based on the following cumulative 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>(Manganese, dichloro [(4aR, 13aR, 17aR, 21aR)-1, 2, 3, 4, 4a, 5, 6, 12, 13, 13a, 14, 15, 16, 17, 17a, 18, 19, 20, 21, 21a-eicosahydro-11, 7-nitrilo-7H-dibenzo[ b,h] [1,4,7,10] tetraazacycloheptadecine-&#954;N5, &#954;N13, &#954;N18, &#954;N21, &#954;N22]-)</p>
Active substance(Manganese, dichloro [(4aR, 13aR, 17aR, 21aR)-1, 2, 3, 4, 4a, 5, 6, 12, 13, 13a, 14, 15, 16, 17, 17a, 18, 19, 20, 21, 21a-eicosahydro-11, 7-nitrilo-7H-dibenzo[ b,h] [1,4,7,10] tetraazacycloheptadecine-κN5, κN13, κN18, κN21, κN22]-)
Medicine Name
Disease/conditionPrevention of oral mucositis in head and neck cancer patients undergoing radiation therapy
Date of decision30/01/2008
OutcomePositive
Orphan decision numberEU/3/07/522

Review of designation

Sponsor’s contact details

Celtic Bio-Pharma Services Ltd
Sheridan House, St Ann's Road
Harrow
Middlesex HA1 1JU
United Kingdom
Tel. +44 (0)2089 972988
Fax +44 (0)2089 972988
E-Mail: gmcgettigan@celticbio.net

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.