EU/3/10/776

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Orphan designation

On 20 September 2010, orphan designation (EU/3/10/776) was granted by the European Commission to Prima Biomed Europe Ltd, United Kingdom, for autologous dendritic cells pulsed with recombinant human-fusion protein (mucin 1 − glutathione S transferase) coupled to oxidised polymannose for the treatment of ovarian cancer.

The sponsorship was transferred to Prima Biomed GmbH, Germany, in February 2012.

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

What is ovarian cancer?

Ovarian cancer is cancer of the ovaries (two organs in the female reproductive system that produce eggs). Most ovarian cancers occur in women over the age of 50 years. Due to the absence of symptoms in the early stages of the disease, the majority of patients are diagnosed when the cancer has spread locally or to other parts of the body.

Ovarian cancer is a life-threatening disease that is associated with poor long-term survival.

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

At the time of designation, ovarian cancer affected approximately 3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 152,000 people*, and is below the threshold 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. At the time of designation, this represented a population of 506,300,000 (Eurostat 2010).

What treatments are available?

At the time of designation, several medicines were authorised in the EU for the treatment of ovarian cancer. The choice of treatment depended mainly on how advanced the disease was. Treatments included surgery and chemotherapy (medicines to treat cancer).

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with ovarian cancer because it works in a different way to existing treatments and because early studies show that it might improve the treatment of patients with this condition, particularly patients whose disease has come back after previous treatment. 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?

Autologous dendritic cells pulsed with recombinant human-fusion protein (mucin 1 − glutathione S transferase) coupled to oxidised polymannose is a cancer vaccine. It is expected to work by activating the patient’s immune system (the body’s natural defences) so that it attacks and kills the cancer cells.

To prepare this medicine, immune system cells called ‘dendritic cells’ are extracted from the patient’s blood. The cells are then incubated (mixed) with a ‘fusion protein’, which is then absorbed into the cells. The fusion protein contains a fragment of mucin 1, a protein that is found in large amounts on the surface of cancer cells but is rarely found on normal cells. When the dendritic cells are injected back into the patient, the immune system is expected to recognise the mucin 1 as ‘foreign’. This is expected to stimulate an immune response against mucin 1, resulting in the immune system attacking the cancerous cells in the body.

What is the stage of development of this medicine?

The effects of autologous dendritic cells pulsed with recombinant human-fusion protein (mucin 1 − glutathione S transferase) coupled to oxidised polymannose 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 ovarian cancer were ongoing.

At the time of submission, this medicine was not authorised anywhere in the EU for ovarian cancer 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 2 June 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>Autologous dendritic cells pulsed with recombinant human-fusion protein (mucin 1 - glutathione S transferase) coupled to oxidised polymannose</p>
Active substanceAutologous dendritic cells pulsed with recombinant human-fusion protein (mucin 1 - glutathione S transferase) coupled to oxidised polymannose
Medicine Name
Disease/conditionTreatment of ovarian cancer
Date of decision20/09/2010
OutcomePositive
Orphan decision numberEU/3/10/776

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

Prima Biomed GmbH
Deutscher Platz 5a 
04103 Leipzig 
Germany
Tel. +49 30 887 168 43
Fax +49 30 690 886 66
E-mail: matt.lehman@primabiomed.com.au

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.