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Orphan designation

On 25 July 2006, orphan designation (EU/3/06/386) was granted by the European Commission to Dr Andreas Kluge, Germany, for 4-[123I] iodo-L-phenylalanine for the diagnosis of glioma.
The sponsorship was transferred to Therapeia GmbH & Co. KG, Germany, in February 2008.

What is glioma?

Glioma is a type of brain tumour that begins in ‘glial’ cells (the cells that surround and support nerve cells). Patients with gliomas can suffer from different symptoms, depending on the part of the brain where the tumour develops. Glioma is a life-threatening disease.

What is the estimated number of patients at risk of developing the condition?

At the time of designation gliomas affected 1.5 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 70,000 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 25), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).

What are the methods of diagnosis available?

Diagnosis for glioma depends on a number of factors and encompasses several methods using different equipment. Diagnosis includes certain contrast agents used for magnetic resonance imaging. Different methods to diagnose gliomas were authorised in the Community at the time of submission of the application for orphan designation.
Satisfactory argumentation has been submitted by the sponsor to justify that 4-[123I] iodo-L-phenylalanine might be of potential significant benefit for the diagnosis of glioma because it might improve the diagnosis of the patients as it can be taken up by malignant glioma cells and retained in the tumours for up to 24 hours. These features may allow improved visualisation and diagnosis of gliomas. 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 medicinal product expected to work?

The product is a radioactive element (123I) attached to an amino acid, L-phenylalanine. Iodine[123I] can give off radiation that can be detected by imaging methods from outside the body. It is expected that 4-[123I] iodo-L-phenylalanine accumulates in the glioma cancer cells and help visualising the tumour tissue with the radiation produced by the attached radioactive iodine.

What is the stage of development of this medicinal product?

The evaluation of the effects of 4-[123I] iodo-L-phenylalanine in experimental models was ongoing.
At the time of submission of the application for orphan designation, clinical trials in patients with glioma were ongoing.
4-[123I] iodo-L-phenylalanine was not authorised anywhere worldwide for glioma or designated as orphan medicinal product elsewhere for this condition, at the time of submission.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 15 June 2006 a positive opinion recommending the grant of the above-mentioned 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>4-[123I] Iodo-L-phenylalanine</p>
Active substance4-[123I] Iodo-L-phenylalanine
Medicine Name
Disease/conditionDiagnosis of glioma
Date of decision25/07/2006
Orphan decision numberEU/3/06/386

Review of designation

Sponsor’s contact details:

Therapeia GmbH & Co.KG
Veilchenweg 38
01326 Dresden
Telephone: +49 351 214 44 12
Telefax: +49 351 214 44 15

Patients’ associations contact points:

Ligue Nationale Contre le Cancer
14 Rue Corvisart
75013 Paris
Telephone: +33 1 53 55 24 00
Telefax: +33 1 43 36 91 10

Deutsche Krebshilfe e. V.
Buschstr. 32
53113 Bonn
Telephone: +49 2 287 29 900
Telefax: +49 2 287 29 90 11

Macmillan Cancer Support (merged with CancerBACUP)
3 Bath Place
Rivington Street
United Kingdom
Telephone: +44 20 76 96 90 03
Switchboard open during office hours, Mon–Fri, 9am–Noon and 2pm–4.45pm
Telefax: +44 20 76 96 90 02