EU/3/08/529 - orphan designation for treatment of visceral leishmaniasis

tretazicar

Overview

On 4 February 2008, orphan designation EU/3/08/529 was granted by the European Commission to Morvus Technology Limited, United Kingdom, for tretazicar for the treatment of visceral leishmaniasis.

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

What is visceral leishmaniasis?

Leishmania are parasites, which are transmitted to humans through the bite of sandflies. In Europe, the disease is found mostly in the southern parts of Europe; it can also be found much more frequently in tropical regions of the world. Following the bite of the sandfly, the parasite is introduced into the skin where it is taken up by some cells of the body's defence system. The parasite then multiplies in these cells, spreads through the body and causes infection. The most severe form of leishmaniasis, so-called visceral leishmaniasis, is caused by parasite infections in the cavities of the body where the major organs are located (most often the abdomen). Patients already with another disease (such as HIV) that suppresses their own defense system are more sensitive to infection by leishmania.

Visceral leishmaniasis is a chronically debilitating and life-threatening condition.

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

At the time of designation, visceral leishmaniasis affected approximately 0.1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 5,000 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).

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* 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. At the time of designation, this represented a population of 502,800,000 (Eurostat 2008).

What treatments are available?

Several medicinal products were authorised for the condition in the Community at the time of submission of the application for orphan designation. Although a significant proportion of patients respond to the initial therapy, many of the immune-suppressed patients tend to get the leishmania infection back and will then not respond well to the therapy.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that the medicinal product might be of potential significant benefit for the treatment of visceral leishmaniasis, particularly because it may 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.

How is this medicine expected to work?

Tretazicar is a product that belongs to a family of products used in the treatment of cancer because of its capacity to kill cells. Tetrazicar is active only after it is transformed inside cells. This is done by an enzyme that is not found in human cells, therefore the damaging activity of tretazicar cannot be seen in humans. However, the enzyme that activates tretazicar is present in leishmania cells where the product can be transformed and be active. Tretazicar kills infected cells by binding to the genetic material (DNA) of the cells and creating bonds that disrupt the genetic material and its function, finally resulting in the death of the infected cell.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, clinical trials in patients with visceral leishmaniasis had been initiated.

The medicinal product was not marketed anywhere worldwide for visceral leishmaniais or designated as an 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 January 2008 recommending the granting of this 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;
• 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 are considered for designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy will be necessary before this product can be granted a marketing authorisation.

EU/3/08/529: Public summary of positive opinion for orphan designation of tretazicar for the treatment of visceral leishmaniasis

Adopted Reference Number: EMEA/COMP/40892/2008 Rev. 1

Key facts

• Active substance:
  tretazicar
• Intended use:
  Treatment of visceral leishmaniasis
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/08/529
• Date of designation:
  04/02/2008
• Sponsor:

  Morvus Technology Limited; Llanvetherine Court; Llanvetherine; Abergavenny; NP7 8NL; United Kingdom; Tel. + 44 (0)1558 668111; E-mail: [e-mail]