EU/3/06/400

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Orphan designation

On 28 August 2006, orphan designation (EU/3/06/400) was granted by the European Commission to Biomedica Life Sciences S.A., Greece, for metastable technetium 99 [99mTc] demogastrin 2 for the diagnosis of medullary thyroid carcinoma.

What is medullary thyroid carcinoma?

Thyroid cancer is a disease in which cancer (malignant) cells are found in certain tissues of the thyroid. The thyroid is a gland in the neck that is composed of mainly two different cell types: the follicular and parafollicular cells. The so-called follicular cells help to concentrate iodine and produce thyroid hormones. These hormones are important for the body growth and metabolism. The parafollicular cells produce a hormone called calcitonin that diminishes the calcium level in the blood. Depending on the type of cell in which the cancer cells originate, different types of thyroid cancer exist.

Medullary thyroid carcinoma is originating from the parafollicular cells (also called C cells), and represents only 5-9% of all thyroid cancers. Signs of cancer are difficult to detect in early stages of the disease and is often limited to a single local swelling of the thyroid gland which is not painful but can be felt by touching. Patients are frequently diagnosed when the disease has spread locally giving symptoms such as shortness of breath, difficulties in swallowing or changes in the voice. Some patients may have severe diarrhoea as a first sign of the disease.
Medullary thyroid carcinoma is a life-threatening disease.

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

At the time of designation, medullary thyroid carcinoma affected approximately 0.7 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 32,000 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 based on data from the European Union (EU 25), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).

What methods of diagnosis are available?

Medullary thyroid carcinoma is diagnosed by elevated levels of calcitonin in the blood and fine needle aspiration of the tumour (cells are sucked out of the tumour through a fine needle to be studied under a microscope). Sometimes magnetic resonance imaging (MRI) and computer tomography (CT) are used to visualise the location of the tumour or tumours, if the disease has spread.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that metastable technetium 99 [99mTc] demogastrin 2 might be of potential significant benefit for the diagnosis of medullary thyroid carcinoma, mainly because it may provide a tool to detect micrometastases (tiny tumours that have spread to other parts of the body). This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

Metastable technetium 99 [99mTc] demogastrin 2 is structurally similar to a part of an endogenous (naturally occurring in the body) protein called gastrin. On the surface of medullary thyroid carcinoma cells there are specific structures (receptors) that bind gastrin. Metastable technetium 99 [99mTc] demogastrin 2 binds to these structures and, because this product has a radioactive part ([99mTc]), its spreading in the body can be detected by imaging methods from the outside. By doing this, it is hoped that small metastases can be found.

What is the stage of development of this medicine?

The effects of metastable technetium 99 [99mTc] demogastrin 2 were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with medullary thyroid carcinoma were ongoing.

Metastable technetium 99 [99mTc] demogastrin 2 was not authorised anywhere worldwide for the diagnosis of medullary thyroid carcinoma, nor 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 July 2006 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;
  • and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

Key facts

Product details for <p>Metastable technetium 99 [99mTc] demogastrin 2</p>
Active substanceMetastable technetium 99 [99mTc] demogastrin 2
Medicine Name
Disease/conditionDiagnosis of medullary thyroid carcinoma
Date of decision28/08/2006
OutcomePositive
Orphan decision numberEU/3/06/400

Review of designation

Sponsor’s contact details:

Biomedica Life Sciences S.A.
2 Tenedou Street
152 35 Vrilissia
Athens
Greece
Telephone: +30 210 689 98 01
Telefax: +30 210 68 99 809
E-mail: info@biomedica.gr

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.