EU/3/08/608

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Orphan designation

On 6 February 2009, orphan designation (EU/3/08/608) was granted by the European Commission to Immunomedics GmbH, Germany, for Yttrium (90Y)-DOTA-radiolabelled humanized monoclonal antibody against mucin 1 for the treatment of pancreatic cancer.

What is pancreatic cancer?

Pancreatic cancer is a cancer of the pancreas, an organ that lies close to the stomach. The pancreas has two functions: producing a juice that helps with the digestion of food, and producing hormones such as insulin. About 95% of pancreatic cancers affect the cells that make the pancreatic juice. These are called adenocarcinomas. Pancreatic cancer is a severe and life-threatening disease.

What is the estimated number of patients affected by the pancreatic cancer?

At the time of designation pancreatic cancer affected approximately 1.3 in 10,000 people in the European Union (EU) *. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP). This is below the threshold for orphan designation which is 5 in 10,000. This is equivalent to a total of around 65,000 people.

*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 27), Norway, Iceland and Liechtenstein. This represents a population of 502,282,000 (Eurostat 2008).

What treatments are available?

At the time of submission of the application for orphan drug designation, several medicines were authorised for the treatment of pancreatic cancer in the European Union. The choice of treatment for pancreatic cancer depends on several factors, including the stage of the disease. Treatments may include surgery, radiotherapy (treatment with radiation), and chemotherapy (medicines used to kill cancer cells). Satisfactory argumentation has been submitted by the sponsor to justify the assumption that Yttrium (90Y)-DOTA-radiolabelled humanized monoclonal antibody against mucin 1 might be of potential significant benefit for the treatment of pancreatic cancer because it has a new mechanism of action which may lead to a better overall outcome for the patients. 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?

Mucin 1 is a protein found on the membrane of many types of cells. However, abnormally high levels or changes in its structure have been associated with some types of cancer and mucin 1 is highly expressed in pancreatic cancer cells. This medicinal product contains an antibody that specifically binds to mucin 1 and is labelled with radioisotope Yttrium (90Y)-DOTA.

Antibodies are proteins used by the immune system to identify and neutralize foreign proteins expressed by bacteria or viruses known as antigens. Antigens and antibodies have a lock-and-key relationship; an antibody can specifically recognize and bind only one antigen. As medicinal products, antibodies can be used to identify specific antigens expressed only on cells of interest. This medicinal product is expected to recognise mucin 1 on pancreatic cancer cells, bind on them and deliver the radioisotope on the tumour. Radioisotopes are used to destroy cancer cells. The antibody is expected to help identify pancreatic cancer cells expressing mucin-1, and once the product is bound, Yttrium (90Y)-DOTA is expected to destroy them.

What is the stage of development of this medicine?

The effects of Yttrium (90Y)-DOTA-radiolabelled humanized monoclonal antibody against mucin 1 have been evaluated in experimental models.

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

At the time of submission, Yttrium (90Y)-DOTA-radiolabelled humanized monoclonal antibody against mucin 1 was not authorised anywhere in the world for pancreatic cancer. Orphan designation of Yttrium (90Y)-DOTA-radiolabelled humanized monoclonal antibody against mucin 1 had been granted in the United States on 29 January 2004 for pancreatic cancer.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 10 December 2008 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>Yttrium (<sup>90</sup>Y)-DOTA-radiolabelled humanized monoclonal antibody against mucin 1</p>
Active substanceYttrium (90Y)-DOTA-radiolabelled humanized monoclonal antibody against mucin 1
Medicine Name
Disease/conditionTreatment of pancreatic cancer
Date of decision05/02/2009
OutcomePositive
Orphan decision numberEU/3/08/608

Review of designation

Sponsor’s contact details:

Immunomedics GmbH
69 Otto-Röhm Straße
64293 Darmstadt
Germany
Telephone: + 49 61 51 66 715 66
Telefax: + 49 6151 66 715 77
E-mail: europe@immunomedics.com

Patients’ associations contact points:

Ligue Nationale Contre le Cancer
14 Rue Corvisart
75013 Paris
France
Telephone: +33 1 53 55 24 00
Telefax: +33 1 43 36 91 10
E-mail: ligue@ligue-cancer.net

Macmillan Cancer Support (merged with CancerBACUP)
3 Bath Place
Rivington Street
London
EC2A 3JR
United Kingdom
Telephone: +44 20 7696 9003
Switchboard open during office hours, Mon–Fri, 9am–Noon and 2pm–4.45pm
Telefax: +44 20 7696 9002

Deutsche Krebshilfe e.V.
Buschstr. 32
53113 Bonn
Germany
Telephone: +49 2 287 29 900
Telefax: +49 2 287 29 90 11
E-mail: deutsche@krebshilfe.de