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Orphan designation

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in January 2011 on request of the sponsor.

On 29 November 2007, orphan designation (EU/3/07/512) was granted by the European Commission to MedImmune Oncology, Inc., Netherlands, for 17-(allylamino)-17-demethoxygeldanamycin hydroquinone hydrochloride for the treatment of malignant gastrointestinal stromal tumours.

The sponsorship was transferred to AstraZeneca AB, Sweden, in June 2008 and subsequently to Voising Consulting S.A.R.L., France, in April 2009.

What are malignant gastrointestinal stromal tumours?

Malignant gastrointestinal stromal tumours (GIST) belong to a special group of gastro-intestinal tumours, called sarcomas. Sarcomas may occur also in other soft tissues of the body; they are cancers of the tissues that support, surround and protect the organs. The cause of GIST is largely unknown. GIST are most common in the stomach (60-70%), followed by the small intestine (20-30%), and then the colon and rectum (5%). GIST occur predominantly in middle-aged and older persons, and are considered as a life-threatening condition.

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

At the time of designation, GIST affected approximately 1.3 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 65,000 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 498,000,000 (Eurostat 2006).

What treatments are available?

The current methods of treatment of GIST are surgical removal of the tumour, and treatment with two (authorised) medicinal products, which belong to a group of substances called tyrosine kinase inhibitors. These products are aimed at slowing down and potentially stopping tumour growth.
17-(allylamino)-17-demethoxygeldanamycin hydroquinone hydrochloride could be of potential significant benefit for the treatment of malignant GIST, mainly because it could act in a different way to currently authorised products, and thus might improve the long-term outcome of 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?

17-(allylamino)-17-demethoxygeldanamycin hydroquinone hydrochloride acts by blocking the function of a protein (a chemical substance within the cells) called HSP90. This protein is involved in the growth and division processes of the cells; by blocking its action, the medicinal product is expected to slow down or stop the growth of GIST.

What is the stage of development of this medicine?

The effects of 17-(allylamino)-17-demethoxygeldanamycin hydroquinone hydrochloride were evaluated in experimental models.

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

The medicinal product was not authorised anywhere in the world for the treatment of GIST, or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 10 October 2007 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>17-(Allylamino)-17-demethoxygeldanamycin, hydroquinone hydrochloride</p>
Active substance17-(Allylamino)-17-demethoxygeldanamycin, hydroquinone hydrochloride
Medicine Name
Disease/conditionTreatment of malignant gastrointestinal stromal tumours
Date of decision28/11/2007
Orphan decision numberEU/3/07/512

Review of designation

Sponsor’s contact details:

Voisin Consulting S.A.R.L.
3, rue des Longs Prés
92100 Boulogne
Telephone: +33 1 41 31 83 00
Telefax: +33 1 41 31 83 09

Patients’ associations contact points:

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.