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Orphan designation

On 6 February 2002, orphan designation (EU/3/01/083) was granted by the European Commission to Ark Therapeutics Limited, United Kingdom, for adenovirus-mediated herpes-simplex-virus thymidine-kinase gene for the treatment of high-grade glioma with subsequent use of ganciclovir sodium.

A marketing authorisation application for this medicine (Cerepro) was withdrawn in July 2007.

A second marketing authorisation application for this medicine (Cerepro) was withdrawn in March 2010.

In February 2014, Ark Therapeutics Limited changed name to Finvector Vision Therapies Limited.

The sponsorship was transferred to Gliotherapy Limited, United Kingdom, in June 2016.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

What is high-grade 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 brain tumours are gliomas, which begin in the glial (supportive) tissue. There are several types of gliomas and these are grouped by grade, which refers to some cell characteristics that can be identified with a microscope.

Cells from higher-grade tumours are more abnormal looking and generally grow faster than cells from lower grade tumours. High-grade tumours are more malignant than low grade tumours and are life-threatening.

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

At the time of designation, high-grade glioma affected approximately 0.7 in 10,000 people in the European Union (EU). This was equivalent to a total of around 27,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the 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. At the time of designation, this represented a population of 380,600,000 (Eurostat 2002).

What treatments are available?

Treatment for high-grade gliomas depends on a number of factors and may include surgery, radiotherapy or chemotherapy as well as symptomatic treatments, such as corticosteroids to control the effects of raised intracranial pressure and anticonvulsants to help control seizures, as required.

Methods of treatment of the condition were authorised at the time of submission of the application for orphan designation. The potential for adenovirus-mediated herpes-simplex-virus thymidine-kinase gene to be of potential significant benefit for the treatment of glioma was based on the novel mechanism of action in particular with regards to improved efficacy.

How is this medicine expected to work?

At the end of the surgical operation to remove the tumour, the thymidine-kinase gene is injected into the underlying brain tissue, where it is taken up into the cells. This is followed by systemic administration of the drug ganciclovir, which is a prodrug (a drug which needs to be activated before it can act). The transferred gene expressed in the cells is able to metabolise ganciclovir transforming it into a drug that is toxic for tumour cells that divide. The toxic form of the drug can also spread to neighbouring cells (‘bystander effect’).

What is the stage of development of this medicine?

Clinical studies of the medicinal product in association with ganciclovir are ongoing in adults with high-grade glioma.

The product had not been marketed in any country at the time of submission of the application for orphan designation. In the United States, orphan-drug status had been granted for use with ganciclovir in the treatment of malignant glioma.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 21 November 2001 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 EU) 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>Adenovirus-mediated herpes-simplex-virus thymidine-kinase (HKSV-tk) gene</p>
Active substanceAdenovirus-mediated herpes-simplex-virus thymidine-kinase (HKSV-tk) gene
Medicine Name
Disease/conditionTreatment of high-grade glioma with subsequent use of ganciclovir sodium
Date of decision06/02/2002
Orphan decision numberEU/3/01/083

Review of designation

Sponsor’s contact details:

Gliotherapy Limited
Sanderum House
Oakley Road
Oxfordshire OX39 4TW
United Kingdom
Tel. +44 (0)1353 645 590
Fax +44 (0)1353 880 098

Patients' organisations:

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.