On 12 February 2015, orphan designation (EU/3/15/1439) was granted by the European Commission to Miltenyi Biotec GmbH, Germany, for allogeneic CD4+ and CD8+ T lymphocytes ex vivo incubated with synthetic peptides of the viral antigens of cytomegalovirus, adenovirus and Epstein-Barr virus for the treatment of cytomegalovirus infection following haematopoietic stem cell transplantation.
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 becomes active if the immune system (the body's natural defences) is weakened.
Cytomegalovirus is therefore a risk in patients with weakened immune systems such as patients who have undergone haematopoietic (blood) stem cell transplantation. Patients undergoing blood stem cell transplantation have their immune systems weakened so that the body does not reject the donor cells. A delay in the recovery of the immune system can cause a reactivation of the latent cytomegalovirus.
Cytomegalovirus infection following haematopoietic stem cell transplantation is a life-threatening and long-term debilitating condition that affects different organs and indirectly affects the immune system, increasing the risk of other infections and the risk of the body rejecting the donor cells.
At the time of designation, cytomegalovirus infection following haematopoietic stem cell transplantation affected less than 0.1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 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).
*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 512,900,000 (Eurostat 2015).
At the time of designation, several antiviral medicines were authorised in the EU for the treatment of cytomegalovirus disease in patients with weakened immune systems (cidofovir, foscarnet, ganciclovir, valaciclovir and valganciclovir).
The sponsor has provided sufficient information to show that this medicine might be of significant benefit for the treatment of cytomegalovirus infection following haematopoietic stem cell transplantation as the medicine has been shown to clear the virus and improve survival in patients who did not respond to previous treatment. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is made from immune cells, known as T lymphocytes, from the same donor that supplied the blood stem cells for the transplant. These immune cells are first incubated with proteins that match those from cytomegalovirus. When the medicine is then given to the patients, the immune cells are expected to start attacking the cytomegalovirus in the patient and thereby help cure the patient of the infection.
The effects of the 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 patients with cytomegalovirus infection following haematopoietic stem cell transplantation had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for the condition or designated as an orphan medicinal product elsewhere.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 9 January 2015 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.
Miltenyi Biotec GmbH
Friedrich-Ebert-Str. 68
Moitzfeld
51429 Bergisch Gladbach
Germany
Tel. +49 22048306820
E-mail: biomedicine@miltenyi.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:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: