EU/3/11/941

  • Email
  • Help

Orphan designation

On 11 January 2012, orphan designation (EU/3/11/941) was granted by the European Commission to Diomune, S.L., Spain, for lipopolysaccharide of Ochrobactrum intermedium for the prevention of sepsis in at-risk premature infants of less than or equal to 32 weeks of gestational age.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

What is sepsis in at-risk premature infants of less than or equal to 32 weeks of gestational age?

Sepsis is a severe bacterial infection of the blood, which may occur in newborn babies. Premature babies born eight weeks or more too early (32 weeks of gestational age or less) have a higher risk of sepsis than other babies. This is because their immune system, skin and moist body surfaces are not yet fully developed, and because they need invasive procedures such as mechanical ventilation (using a machine to help them to breathe) or administration of medicines or feeding through a vein, which can put them at risk of infections in neonatal intensive care units.

Sepsis is a life-threatening condition which may cause multiple organ failure. Sepsis is one of the most common causes of death in premature babies born at 32 weeks of gestational age or less.

What is the estimated number of patients at risk of developing the condition?

At the time of designation, the number of premature babies of less than or equal to 32 weeks of gestational age at risk of sepsis was estimated to be approximately 1.5 in 10,000 in the European Union (EU). This was equivalent to a total of around 76,000 babies*, 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. At the time of designation, this represented a population of 509,000,000 (Eurostat 2012).

What methods of prevention are available?

At the time of designation, several antibiotics were used to treat and to prevent sepsis in premature babies.

The sponsor has provided sufficient information to show that lipopolysaccharide of Ochrobactrum intermedium might be of significant benefit for premature babies at risk of sepsis because it works in a different way to existing treatments and may improve the outcome of patients at risk of this condition. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

The medicine is expected to mildly stimulate the immune system (the body’s natural defences) of premature infants because it is very similar to a structure on the surface of bacteria, which may cause sepsis. This structure is recognised as ‘foreign’ by immune cells because it attaches to and activates two receptors called ‘Toll-like receptor 2’ and ‘Toll-like receptor 4’ on the surface of these immune cells. Their activation is expected to stimulate other immune cells to attack bacteria when the premature infant is exposed to them. This is expected to help prevent sepsis.

What is the stage of development of this medicine?

The effects of lipopolysaccharide of Ochrobactrum intermedium 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 sepsis had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for the prevention of sepsis 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 9 November 2011 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>Lipopolysaccharide of <em>Ochrobactrum intermedium</em></p>
Active substanceLipopolysaccharide of Ochrobactrum intermedium
Medicine Name
Disease/conditionPrevention of sepsis in at-risk premature infants of less than or equal to 32 weeks
Date of decision11/01/2012
OutcomePositive
Orphan decision numberEU/3/11/941

Review of designation

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

Sponsor’s contact details:

Diomune S.L.
C/ Felix Boix Nº9 4ºC
28036 Madrid
Spain
Tel. +34 91 806 4656
Fax +34 91 803 1031
E-mail: info@diomune.com

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.