Overview

On 25 May 2018, orphan designation (EU/3/18/2024) was granted by the European Commission to Galephar M/F, Belgium, for itraconazole for the prevention of invasive aspergillosis.

The sponsorship was transferred to Laboratoires S.M.B., Belgium, in May 2020.

Invasive aspergillosis is an infection caused by species of fungi known as Aspergillus. Spores of these fungi are widely present in the air, but normally do not cause disease. In some people, usually when the immune system (the body's natural defences) is weakened, the fungus can attack and invade the lungs and spread throughout the body via the bloodstream, damaging other organs and tissues including the heart, kidney, liver, brain and bones.

Invasive aspergillosis is a life-threatening disease that can be fatal due to damage to the lungs and other organs.

At the time of designation, the number of patients at risk of invasive aspergillosis was estimated to be less than 2 people in 10,000 in the European Union (EU). This was equivalent to a total of fewer than 103,000 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 at risk of developing the condition is estimated and assessed on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 517,400,000 (Eurostat 2018).

At the time of designation, several antifungal medicines were used in the EU for the prevention of invasive aspergillosis, including amphotericin, caspofungin, isavuconazole, itraconazole, posaconazole, voriconazole. These medicines were mostly available for use by injection or by mouth.

The sponsor has provided sufficient information to show that the medicine (itraconazole by inhalation) might be of significant benefit for patients at risk of invasive aspergillosis. Laboratory data showed greater clearance of the infection and higher survival with the medicine given by inhalation than with another antifungal medicine given by mouth. In addition, the inhalation route may offer better safety.

This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Itraconazole has been used to treat fungal infections for several years. It works by blocking certain enzymes in the fungus known as cytochrome p450, thus preventing the formation of ergosterol, which is an important part of fungal cell walls. This ultimately leads to the fungus' death and clearance of the infection.

The effects of the itraconazole have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients at risk of invasive aspergillosis had started.

At the time of submission, itraconazole was not authorised anywhere in the EU for prevention of invasive aspergillosis or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 19 April 2018 recommending the granting of this designation.

  • 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.

EU/3/18/2024: Public summary of opinion on orphan designation: Itraconazole for the prevention of invasive aspergillosis

Reference Number: EMA/272912/2018

English (EN) (113.12 KB - PDF)

First published: Last updated:
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Key facts

Active substance
Itraconazole
Intended use
Prevention of invasive aspergillosis
Orphan designation status
Positive
EU designation number
EU/3/18/2024
Date of designation
Sponsor

Laboratoires S.M.B.
Rue De La Pastorale 26-28
1080 Molenbeek-Saint-Jean
Brussels
Belgium
Tel. +32 2 4114828
E-mail: regulatory@smb.be

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

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:

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