Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in March 2015 on request of the Sponsor.
On 19 February 2014, orphan designation (EU/3/14/1235) was granted by the European Commission to Roche Registration Limited, United Kingdom, for mixture of recombinant human IgG1 monoclonal antibodies against human cytomegalovirus envelope glycoproteins for the prevention of congenital cytomegalovirus infection following primary cytomegalovirus infection.
For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.
Cytomegalovirus is a common virus that usually only causes mild infection, with symptoms such as fever and 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 immune system (its natural defences) is weakened. Active cytomegalovirus disease can affect several organs in the body, such as the eyes, liver, lungs and the gastrointestinal tract (the stomach and intestines) and cause organ failure. If a woman is infected for the first time (primary infection) with cytomegalovirus while pregnant, the virus can pass to the unborn baby (congenital infection) as it does not yet have a very strong immune system. Congenital infection can cause severe disease at birth and lead to growth retardation and brain damage. Congenital cytomegalovirus disease is long-term debilitating and life-threatening.
At the time of designation, the number of patients at risk of congenital cytomegalovirus infection following primary cytomegalovirus infection was less than 1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 51,000 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 28), Norway, Iceland and Liechtenstein. This represents a population of 511,100,000 (Eurostat 2014).
At the time the application was submitted there were no medicines authorised in the EU for the prevention of congenital cytomegalovirus infection following primary cytomegalovirus infection in pregnant women.
The medicine contains a mixture of two antibodies (proteins) that recognise and attach to specific structures on the surface of cytomegalovirus. These structures are part of the mechanism that the virus uses to enter and infect the cells of the body. By attaching to and blocking these structures, the medicine is expected to help prevent the virus from infecting the developing baby, and so prevent congenital cytomegalovirus infection and its consequences.
The effects of this medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with the medicine in pregnant women with primary cytomegalovirus infection had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for the prevention of congenital cytomegalovirus infection following primary cytomegalovirus infection in pregnant women. Orphan designation of the medicine had been granted in the United States for the condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 9 January 2014 recommending the granting of this designation.
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.
Roche Registration Limited
6 Falcon Way
Shire Park
Welwyn Garden City
AL7 1TW
United Kingdom
Tel. +44 (0)1707 362840
Fax +44 (0)1707 377838
E-mail: info.orphan@roche.com
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
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.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: