EU/3/08/529

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.

What is visceral leishmaniasis?

Leishmania are parasites, which are transmitted to humans through the bite of sand flies. 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 will be taken up by some cells of the body’s defence system. The parasite will then multiply in these cells and spread through the body and cause infection. The most severe form of leishmaniasis, the 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 having already another disease (such as HIV) that suppresses their own defense system are more sensitive to infection by the leishmania.

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

What are the methods of treatment 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.

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

Based on the information provided by the sponsor and previous knowledge of the Committee, visceral leishmaniasis was considered to affect approximately 0.1 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 5,000 persons in the total.

* 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 27), Norway, Iceland and Lichtenstein. This represents a population of 498,000,000 (Eurostat 2006). 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?

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 medicinal product?

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

The medicinal product was not marketed anywhere worldwide for visceral leishmaniais 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 January 2008 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.