EU/3/12/1073

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Orphan designation

On 6 December 2012, orphan designation (EU/3/12/1073) was granted by the European Commission to Bayer Pharma AG, Germany, for maytansinoid-conjugated human monoclonal antibody against mesothelin for the treatment of mesothelioma.

What is malignant mesothelioma?

Malignant mesothelioma is a cancer that affects the mesothelial cells (found on the inner linings of the organs), mainly the pleura (the lining of the lungs) and the peritoneum (the lining of the abdominal cavity). It is usually caused by exposure to asbestos. Mesothelioma of the pleura causes difficulty breathing and chest pain, and mesothelioma of the peritoneum causes ascites (a build-up of fluid in the abdomen) and abdominal pain. Because the symptoms are not specific, the cancer is often detected at a late stage.

Malignant mesothelioma is life-threatening because it may lead to bowel obstruction or breathing problems and lung infections. Patients have very poor survival, only living for a year, on average, after diagnosis.

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

At the time of designation, malignant mesothelioma affected not more than 0.3 in 10,000 people in the European Union (EU)*. This is equivalent to a total of not more than 15,000 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 506,300,000 (Eurostat 2011).

What treatments are available?

At the time of designation, the main treatment for malignant mesothelioma was surgery followed by chemotherapy (medicines to treat cancer) or radiotherapy (treatment with radiation). If the disease was too advanced for surgery, chemotherapy alone was used. One medicine, pemetrexed, was authorised in the EU for the treatment of malignant pleural mesothelioma.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with malignant mesothelioma because it acts in a different way to existing treatments by targeting the mesothelioma cells which might improve the treatment of patients when used alone or in combination with other medicines. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain orphan status.

How is this medicine expected to work?

This medicine is made up of two components:

  • a monoclonal antibody, a type of protein that has been designed to recognise and attach to a naturally occurring protein called ‘mesothelin’. Mesothelin is a protein that is found in large amounts on the surface of cancer cells, such as the cells in malignant mesothelioma;
  • a maytansinoid, a substance that kills cells by preventing cell division.

This medicine is expected to target mesothelioma cells by attaching to mesothelin. Once the medicine is attached to these cells, it is expected to be taken inside the cells and to release the maytansinoid, which blocks cell division. This is expected to slow down the growth of the mesothelioma.

What is the stage of development of this medicine?

The effects of the medicinal product have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with this medicine in patients with malignant mesothelioma were ongoing.

At the time of submission, the medicinal product was not authorised anywhere in the EU for malignant mesothelioma. Orphan designation had been granted in the United States for mesothelioma.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 November 2012 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>Maytansinoid-conjugated human monoclonal antibody against mesothelin</p>
Active substanceMaytansinoid-conjugated human monoclonal antibody against mesothelin
Medicine Name
Disease/conditionTreatment of malignant mesothelioma
Date of decision06/12/2012
OutcomePositive
Orphan decision numberEU/3/12/1073

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:

Bayer Pharma AG
Muellerstrasse 178,
D-13353 Berlin
Germany
Tel. +49 30 468 1111
Fax +49 30 468 15305
E-mail: bayer-pharma-gra-eu@bayer.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.