EU/3/05/343 - orphan designation for treatment of glioma
Enzastaurin hydrochloride
OrphanHuman
Overview
Please note that this product was withdrawn from the Union Register of orphan medicinal products in May 2024 on request of the Sponsor.
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.
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.
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.
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.
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.
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.
- 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
- Active substance
- Enzastaurin hydrochloride
- Intended use
- Treatment of glioma
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/05/343
- Date of designation
- Sponsor
Orphix Consulting GmbH
Update history
| Date | Update |
|---|---|
| October 2020 | The sponsorship was transferred to Dlrc Pharma Services Limited, Ireland, in October 2020. |
| February 2016 | The sponsorship was transferred to Isabelle Ramirez, Germany, in February 2016. |
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: