EU/3/12/1048

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Orphan designation

On 10 October 2012, orphan designation (EU/3/12/1048) was granted by the European Commission to European Medical Advisory Services Limited, United Kingdom, for A mixture of two allogeneic human pancreatic-cancer cell lines stably transduced with a retroviral vector encoding the murine alpha-(1,3)-galactosyltransferase gene (also known as algenpantucel-L) for the treatment of pancreatic cancer.

What is pancreatic cancer?

Pancreatic cancer is cancer of the pancreas, a small organ that lies behind the stomach. The pancreas has two functions: to produce a juice that helps with the digestion of food, and to produce hormones such as insulin. Due to the absence of symptoms in the early stages of pancreatic cancer, the majority of patients are diagnosed when the cancer has spread locally or to other parts of the body.

Pancreatic cancer is a very severe and life-threatening disease that is associated with shortened life expectancy.

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

At the time of designation, pancreatic cancer affected 1.3 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 66,000 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, several medicines were authorised in the EU for treating pancreatic cancer. The choice of treatment depended on several factors, including how far the disease had advanced.

Treatments included surgery, radiotherapy (treatment with radiation) 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 pancreatic cancer because early studies show that it works in a different way to existing treatments and might improve the outcome of patients with this condition when used together with currently authorised treatments. 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?

The medicine is expected to work as a ‘cancer vaccine’, by activating the patient’s immune system (the body’s natural defences) so that it attacks and kills the cancer cells. The medicine is made of pancreatic cancer cells that have been modified to produce an enzyme called alpha-(1,3)-galactosyltransferase. This enzyme is responsible for the production of substances called alpha-Gal, which are known to trigger an immune response. When the patient is given the vaccine, the patient’s immune system is expected to stimulate an immune response not only against the cancer cells in the vaccine which contain alpha-Gal, but also against the cancer cells in the patients even though they do not contain alpha-Gal.

What is the stage of development of this medicine?

The effects of this medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with pancreatic cancer were ongoing.

At the time of submission, this medicine was not authorised anywhere in the EU for pancreatic cancer. Orphan designation of the medicine had been granted in the United States of America for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 5 September 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>Algenpantucel-L</p>
Active substanceAlgenpantucel-L
Medicine Name
Disease/conditionTreatment of pancreatic cancer
Date of decision10/10/2012
OutcomePositive
Orphan decision numberEU/3/12/1048

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:

European Medical Advisory Services Limited
71 Knowl Piece
Wilbury Way
Hitchin, Herts SG4 0TY
United Kingdom
Telephone: +44 14 62 424400
Telefax: +44 14 62 424426
E-mail: neil.murray@emaspharma.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.