EU/3/09/624

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Orphan designation

On 29 April 2009, orphan designation (EU/3/09/624) was granted by the European Commission to Antigenics Therapeutics Limited, Ireland, for autologous tumour-derived gp96 heat shock protein-peptide complex for the treatment of glioma.

What is glioma?

Glioma is a type of brain tumour that affects the ‘glial’ cells (the cells that surround and support the nerve cells). Patients with glioma can have severe symptoms, but the types of symptoms experienced depend on where the tumour develops in the brain. Symptoms can include headaches, nausea (feeling sick), loss of appetite, vomiting, and changes in personality, mood, mental capacity and concentration. About a fifth of patients with glioma have seizures (fits) for months or years before the disease is diagnosed.

Glioma is a debilitating and life-threatening disease because of the severe damage to the brain that leads to poor long-term survival.

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

At the time of designation, glioma affected approximately 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 50,000 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 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 504,800,000 (Eurostat 2009).

What treatments are available?

At the time of designation, several medicines were authorised for the treatment of glioma in the EU. Treatments for glioma included surgery, radiotherapy (using radiation to kill the cancer cells) and chemotherapy (medicines used to kill cancer cells). Patients also received treatments for the symptoms of glioma, including corticosteroids to reduce pressure within the skull and medicines to prevent fits.

The sponsor has provided sufficient information to show that autologous tumour-derived gp96 heat shock protein-peptide complex might be of significant benefit for the patients because it works in a different way to existing medicines. 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 tumour-derived gp96 heat shock protein-peptide complex is expected to work as a tumour vaccine. It is made up of substances extracted from the patient’s tumour, a protein called ‘gp96 heat shock protein’ and peptides (protein fragments), which form a fingerprint for that tumour.

When the patient is given the vaccine, the heat shock protein and the peptides are now recognised as ‘foreign’ by the immune system (the body’s natural defences), stimulating an immune response. This is expected to lead to the immune system being able to attack and kill the tumour cells and not cells elsewhere in the body.

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 in patients with glioma and other types of cancer were ongoing.

At the time of submission, this medicine was not authorised anywhere in the EU for glioma or designated as 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 4 March 2009 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 Community) 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 tumor-derived gp96 heat shock protein-peptide complex</p>
Active substanceAutologous tumor-derived gp96 heat shock protein-peptide complex
Medicine Name
Disease/conditionTreatment of glioma
Date of decision29/04/2009
OutcomePositive
Orphan decision numberEU/3/09/624

Review of designation

Sponsor’s contact details:

Antigenics Therapeutics Limited
25/28 North Wall Quay
Dublin 1
Ireland
Telephone: +353 1 649 2000
Telefax: +353 1 649 2649
E-mail: orphan@voisinconsulting.com

Patient 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

Deutsche Krebshilfe e. V.
Buschstr. 32
53113 Bonn
Germany
Telephone: +49 2 28/7 29 90–0
Telefax: +49 2 28/7 29 90–11
E-mail: deutsche@krebshilfe.de

Macmillan Cancer Support
3 Bath Place
Rivington Street
London EC2A 3JR
United Kingdom
Telephone: +44 20 7696 9003
Telefax: +44 20 7696 9002

European Cancer Patient Coalition
ECPC Office
Am Rothenanger 1b
85521 Riemerling
Germany
Tel.: +49 89 628 36 807
Fax: +49 89 628 36 808
E-mail: info@ecpc-online.org