EU/3/05/343

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Orphan designation

On 23 December 2005, orphan designation (EU/3/05/343) was granted by the European Commission to Eli Lilly Nederland B.V., The Netherlands, for enzastaurin hydrochloride for the treatment of glioma.

What is glioma?

Tumours that begin in brain tissue are known as primary brain tumours. Primary brain tumours are classified by the type of tissue from which they originate, the most common being gliomas, which begin in the glial (supportive) tissue.
Gliomas represent a potentially debilitating and life-threatening condition. Patients affected by gliomas can suffer from severe symptoms of the nervous system, depending on where in the brain the tumour develops.

What are the methods of treatment available?

Treatment for gliomas depends on a number of factors and encompasses several methods such as surgery, radiotherapy (using high-dose x-rays or other high-energy rays to kill cancer cells) or chemotherapy (using drugs to kill cancer cells), as well as some symptomatic treatments. Symptomatic treatments include certain steroid hormones (corticosteroids) to control the effects of raised pressure within the skull, and medication to help control seizures, as required. Methods of treatment of glioma were authorised in the Community at the time of submission of the application for orphan designation.
Enzastaurin hydrochloride might be of potential significant benefit for the treatment of gliomas because it might improve the long-term outcome of the patients. The assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

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

According to the information provided by the sponsor, glioma was considered to affect about 46,000 persons in the European Union.

*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 Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

How is this medicinal product expected to act?

Cancer cells need to replicate and produce a network of new blood vessels in order to grow. Enzastaurin hydrochloride blocks (inhibits) the function of certain proteins such as protein kinase pathways that have been shown to be involved in the progression of some types of cancer, including glioma. The theory is that enzastaurin hydrochloride, through its interference with the function of these proteins, will prevent the glioma from growing by preventing tumour cell replication and development of new blood vessels and by inducing tumour cell death.

What is the stage of development of this medicinal product?

The evaluation of the effects of enzastaurin hydrochloride in experimental models is ongoing.
At the time of submission of the application for orphan designation, clinical trials in patients with glioma were ongoing.
Enzastaurin hydrochloride 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 10 November 2005 a positive opinion recommending the grant of the above-mentioned designation.

Opinions on orphan medicinal products designations are based on the following cumulative criteria:
  • the seriousness of the condition, 
  • the existence or not of alternative methods of diagnosis, prevention or treatment and 
  • either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments.

Designated orphan medicinal products are still investigational products which were considered for 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 will be necessary before this product can be granted a marketing authorisation.

Key facts

Product details for <p>Enzastaurin hydrochloride</p>
Active substanceEnzastaurin hydrochloride
Medicine Name
Disease/conditionTreatment of glioma
Date of decision23/12/2005
OutcomePositive
Orphan decision numberEU/3/05/343

Review of designation

Sponsor’s contact details:

Eli Lilly Holdings Limited
European Operations
Sunninghill Road
Windlesham
Surrey
GU20 6PH
United Kingdom
Telephone: +44 12 76 48 30 00
Telefax: +44 12 76 48 49 21

Patients’ associations contact points:

Cancer research UK
P.O. Box 123 Lincln's Inn Fields
WC2A 3PX
London
United Kingdom
Telephone : +44 20 72 42 02 00
Telefax: +44 20 72 69 31 00

Ligue Nationale Contre le Cancer
13 Av. de la Grande Armee
75116 Paris
France
Telephone: +33 1 45 00 00 17
Tefefax: +33 1 45 00 63 06
E-mail: ligue@ligue-cancer.net

A.I.T.C. - Associazione Italiana per i Tumori Cerebrali
Istituto Neurologico Nazionale C. Besta
Laboratorio di Neuroncologia e Terapia Genica
Via Celoria 11
20133 Milano
Italy
Telephone: +39 02 23 94 453
Telefax: +39 02 26 68 16 88
E-mail: info@tumoricerebrali.it