EU/3/13/1133 - orphan designation for treatment of cytomegalovirus disease in patients with impaired cell-mediated immunity

Maribavir
OrphanHuman

Overview

On 7 June 2013, orphan designation (EU/3/13/1133) was granted by the European Commission to ViroPharma SPRL, Belgium, for maribavir for the treatment of cytomegalovirus disease in patients with impaired cell-mediated immunity.

The medicinal product has been authorised in the EU as Livtencity since 09 November 2022.

Cytomegalovirus is a common virus that usually only causes mild infection such as a sore throat. Most people get infected at some stage during their lifetime but are very often unaware of it. After infection, the virus usually remains in the body in a 'latent' (inactive) state and only becomes active if the body's immunity, specifically its cell-mediated immunity, is weakened.

Cell-mediated immunity is a defence mechanism where specialised cells called T lymphocytes directly neutralise viruses. In people with weakened cell-mediated immunity, such as transplant patients receiving immunosuppressant treatment (medicines that reduce the activity of the immune system), cytomegalovirus is not neutralised and cause severe infection.

Cytomegalovirus disease in patients with impaired cell-mediated immunity is long-term debilitating and life-threatening because of the complications it causes, such as inflammation of the eyes, lungs, liver and digestive tract, as well as reduced survival of transplanted organs or tissues in transplant patients.

At the time of designation, cytomegalovirus disease in patients with impaired cell-mediated immunity affected approximately 2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 102,000 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 (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 509,000,000 (Eurostat 2013).

At the time of designation, several antiviral medicines were authorised in the EU for the treatment of cytomegalovirus disease in patients with impaired cell-mediated immunity (cidofovir, foscarnet, ganciclovir, valaciclovir and valganciclovir).
The sponsor has provided sufficient information to show that maribavir might be of significant benefit for the treatment of cytomegalovirus disease in patients with impaired cell-mediated immunity because there is evidence that it may be effective in patients with cytomegalovirus disease resistant to existing antiviral medicines. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

When cytomegalovirus multiplies during an infection, its genetic material (DNA) is replicated and packaged into small protein shells, giving rise to newly formed viruses that can proceed to infect other cells. Maribavir is thought to block the action of an enzyme of the virus called UL97 kinase, which is involved in packaging the DNA in the protein shells and releasing new viruses. By blocking the enzyme, the medicine is expected to prevent viruses from reaching maturity, so that no new infectious viruses can be produced.

The effects of maribavir have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with maribavir in patients with cytomegalovirus disease and impaired cellular immunity were ongoing.

At the time of submission, maribavir was not authorised anywhere in the EU for treatment of cytomegalovirus disease. Orphan designation of maribavir was previously granted in the EU for prevention of cytomegalovirus disease in patients at risk. Orphan designations had also been granted in the United States for prevention and treatment of cytomegalovirus disease in at-risk patients.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 17 April 2013 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.

Key facts

Active substance
Maribavir
Intended use
Treatment of cytomegalovirus disease in patients with impaired cell-mediated immunity
Orphan designation status
Positive
EU designation number
EU/3/13/1133
Date of designation
Sponsor

Takeda Pharmaceuticals International AG Ireland Branch

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Livtencity at the time of marketing authorisation, and confirmed that the orphan designation should be maintained. More information is available in the Livtencity : Orphan maintenance assessment report (initial authorisation).

Update history

DateUpdate
October 2022The sponsor's address was updated in October 2022.
September 2021The sponsorship was transferred to Takeda Pharmaceuticals International AG Ireland Branch.
March 2016The sponsorship was transferred to Shire Pharmaceuticals Ireland Limited, Ireland.
February 2016ViroPharma SPRL changed name to Shire Services BVBA.
February 2009The sponsorship was transferred to ViroPharma SPRL, Belgium.

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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