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Orphan designation

On 14 October 2016, orphan designation (EU/3/16/1744) was granted by the European Commission to BrainRepair UG (haftungsbeschränkt), Germany, for autologous mononuclear cells derived from human cord blood for the treatment of periventricular leukomalacia.

What is periventricular leukomalacia?

Periventricular leukomalacia is a type of brain damage that occurs in premature babies because of a lack of oxygen or blood in parts of the brain (‘white matter’). The condition is not usually noticeable at birth, but signs of brain damage may start to show in early childhood.

Periventricular leukomalacia is life-threatening and debilitating in the long term because of the risk of the child developing problems with learning, sight, behaviour and movement.

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

At the time of designation, periventricular leukomalacia affected approximately 0.3 in 10,000 people per year in the European Union (EU). This was equivalent to a total of around 15,000 people per year*, and is below the ceiling for orphan designation. This isbased 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 513,700,000 (Eurostat 2016).

What treatments are available?

At the time of the orphan designation there were no satisfactory treatments authorised for periventricular leukomalacia in the EU.

How is this medicine expected to work?

This medicine is a type of advanced therapy medicine called a ‘tissue engineered product’. This is a type of medicine containing cells or tissues that have been manipulated so that they can be used to repair, regenerate or replace tissue.

The medicine is made of ‘mononuclear cells’, which include immune cells and stem cells (cells that can develop into different types of cell), from the blood in the baby’s umbilical cord. At birth, the cord blood is collected and the mononuclear cells are harvested and stored, so that they are ready to be injected back into the baby should periventricular leukomalacia occur. When injected, the mononuclear cells are expected to move to the oxygen-poor damaged brain region, where they release cytokines (messenger molecules of the immune system) and other substances, which in turn are expected to improve survival of brain cells. This is expected to improve the symptoms of the condition.

What is the stage of development of this medicine?

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 periventricular leukomalacia had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for periventricular leukomalacia 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 8 September 2016 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:
  • 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

Product details for <p>Autologous mononuclear cells derived from human cord blood</p>
Active substanceAutologous mononuclear cells derived from human cord blood
Medicine Name
Disease/conditionTreatment of periventricular leukomalacia
Date of decision14/10/2016
Orphan decision numberEU/3/16/1744

Review of designation

Sponsor’s contact details

BrainRepair UG (haftungsbeschränkt)
Universitätsstr. 140
44799 Bochum
Tel. +49 234 5881 960
Fax +49 234 5881 9619

Patients’ organisations

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.