EU/3/06/414

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Orphan designation

On 3 November 2006, orphan designation (EU/3/06/414) was granted by the European Commission to Fresenius Biotech GmbH, Germany, for catumaxomab for the treatment of gastric cancer.

In January 2014, Fresenius Biotech GmbH changed name to Neovii Biotech GmbH.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

What is gastric cancer?

Cancer tumours that originate in the stomach are called gastric cancers. Most gastric cancers are adenocarcinomas, so called because they start in the glandular cells that line the inside of the stomach (the mucosa). These cancers are usually first discovered and diagnosed in the affected patients at an advanced stage of the disease when the tumours have spread to surrounding organs in the abdomen or other parts of the body. Symptoms of gastric cancer include loss of appetite, vomiting, weight loss, bleeding in the stomach and subsequent anaemia (fewer red blood cells in the blood than normal). Gastric cancer is chronically debilitating and life threatening.

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

At the time of designation, gastric cancer affected approximately 3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 141,000 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 25), Norway, Iceland and Liechtenstein. At the time of designation, this represented a population of 468,900,000 (Eurostat 2006).

What treatments are available?

The choice of the treatment of gastric cancers depends on several factors, including the stage of the disease. Treatments may include surgery, radiation therapy, and chemotherapy (using drugs to kill cancer cells). There were several medicinal products authorised for the treatment of gastric cancer in the Community at the time of orphan status designation.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that catumaxomab might be of potential significant benefit for the treatment of gastric cancer mainly because it has a new mechanism of action and may be used in combination with other treatments and because it may provide a major contribution to patient care. 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?

Catumaxomab is an antibody designed to bind specifically on three different types of cells: tumour cells, T cells and accessory immune cells (e.g. macrophages, dendritic cells and natural killer cells). By binding to the three types of cells, the product is expected to induce activation of T cells and accessory cells against cancer cells. This is thought to lead to elimination of the cancer cells.

What is the stage of development of this medicine?

The effects of catumaxomab were evaluated in experimental models. At the time of submission of the application for orphan designation, clinical trials in patients with gastric cancer were ongoing.

Catumaxomab was not authorised anywhere worldwide for the treatment of gastric cancer 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 4 October 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.

Name Language First published Last updated
EU/3/06/414: Public summary of positive opinion for orphan designation of catumaxomab for the treatment of gastric cancer (English only) 23/02/2009 07/02/2014

Key facts

Product details for <p>Catumaxomab</p>
Active substanceCatumaxomab
Medicine Name
Disease/conditionTreatment of gastric cancer
Date of decision02/11/2006
OutcomePositive
Orphan decision numberEU/3/06/414

Review of designation

Sponsor’s contact details:

Neovii Biotech GmbH
Am Haag 6-7
D-82166 Graefelfing
Germany
Tel. +49 89 89 88 88 260
Fax +49 89 89 88 88 17
E-mail: regulatory@fresenius-biotech.com

Patients’ association(s) contact point(s):

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.