EU/3/05/294

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Orphan designation

On 16 June 2005, orphan designation (EU/3/05/294) was granted by the European Commission to Biotec Pharmacon ASA, Norway, for soluble yeast beta-1,3/1 6- glucan 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 highdose x-rays or other high-energy rays to kill cancer cells. Radiation therapy will not only kill the tumour cells but will also cause some degree of injury to the surrounding tissue. Radiation therapy given to tumours located in the head and neck region might thus harm the rapidly dividing inner lining (mucosa) of the mouth. This complication of radiation therapy is called oral mucositis. The condition is chronically debilitating, resulting in severe pain and dryness of the mouth, which leads to difficulties in swallowing.

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 not more than 2 people in 10,000 in the European Union (EU)*. This is equivalent to a total of not more than 92,000 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 25), Norway, Iceland and Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004).

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?

It is not yet fully understood how soluble yeast beta-1,3/1,6- glucan exerts a direct or indirect protection against the negative effects induced by the radiation therapy. It seems that it might act at different levels, by enhancing, for example, the wound healing after the oral lesion, and by acting locally on certain cells within the mucosa, belonging to the body’s defence system (immune competent cells).This might stimulate the immune system against possible infections while reducing the inflammation reaction. 

What is the stage of development of this medicine?

The evaluation of the effects of soluble yeast beta-1,3/1,6-glucan in experimental models is ongoing. 

At the time of submission of the application for orphan designation, clinical trials in head and neck cancer patients receiving radiotherapy were ongoing. 

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 12 May 2005 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>Soluble yeast beta-1,3/1,6- glucan</p>
Active substanceSoluble yeast beta-1,3/1,6- glucan
Medicine Name
Disease/conditionPrevention of oral mucositis in head and neck cancer patients undergoing radiation therapy
Date of decision16/06/2005
OutcomePositive
Orphan decision numberEU/3/05/294

Review of designation

Sponsor’s contact details:

Biotec Pharmacon ASA
Strandgata 3
9008 Tromsø
Norway
Telephone: +47 77 64 89 00
Telefax: +47 77 64 89 01
E-mail: mail@biotec.no

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.