EU/3/10/842

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Orphan designation

On 23 February 2011, orphan designation (EU/3/10/842) was granted by the European Commission to Azanta A/S, Denmark, for nimorazole for the treatment of squamous cell carcinoma of the head and neck in patients undergoing radiotherapy.

What is squamous cell carcinoma of the head and neck?

Squamous cell carcinoma of the head and neck is a type of cancer that starts in the ‘squamous cells’ lining the mouth, nose, throat or larynx (voice box). This type of cancer is often associated with alcohol and tobacco use, and may also be associated with local viral infection. Radiotherapy, the use of radiation to kill cancer cells, is used in about two thirds of patients with this cancer.

Squamous cell carcinoma of the head and neck is a long-lasting, debilitating and life-threatening disease that is associated with poor overall survival.

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

At the time of designation, squamous cell carcinoma of the head and neck in patients undergoing radiotherapy affected less than 4 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 202,000 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 506,500,000 (Eurostat 2010).

What treatments are available?

At the time of designation, treatment of patients with squamous cell carcinoma of the head and neck depended on the site and stage of development of their cancer. Surgery was widely used where possible to remove the tumour, followed by radiotherapy and chemotherapy (medicines to treat cancer), while a number of other medicines were authorised for use in this disease.

The sponsor has provided sufficient information to show that nimorazole might be of significant benefit for patients with squamous cell carcinoma of the head and neck undergoing radiotherapy, because it works in a different way to existing treatments and may enhance the effectiveness of radiotherapy, thus improving the treatment of patients with this condition. 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?

Nimorazole belongs to a group called ‘nitroimidazoles’ that are widely used as anti-infective agents and is already authorised in some EU countries as an antimicrobial agent.

In squamous cell carcinoma of the head and neck, nimorazole is expected to work by acting as a ‘radiotherapy sensitiser’. This means that it is expected to make the cancer cells more sensitive to the effect of radiation, thus enhancing the effect of radiotherapy.

What is the stage of development of this medicine?

The effects of nimorazole have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with nimorazole in patients with squamous cell carcinoma of the head and neck undergoing radiotherapy were ongoing.

At the time of submission, nimorazole was not authorised anywhere in the EU for squamous cell carcinoma of the head and neck in patients undergoing radiotherapy 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 10 November 2010 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>Nimorazole</p>
Active substanceNimorazole
Medicine Name
Disease/conditionTreatment of squamous cell carcinoma of the head and neck in patients undergoing radiotherapy
Date of decision23/02/2011
OutcomePositive
Orphan decision numberEU/3/10/842

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:

Azanta A/S
Tranegaardsvej 20
2900 Hellerup
Denmark
Telephone: +45 70 25 95 45
Telefax: +45 70 25 95 46
E-mail: info@azanta.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.