EU/3/05/335 - orphan designation for late onset sepsis in premature infants of less or equal than 32 weeks of gestational age

Human Staphylococcus aureus immunoglobulin / human Staphylococcus epidermidis polyclonal immunoglobulin

Overview

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in March 2008 on request of the sponsor.

On 14 December 2005, orphan designation (EU/3/05/335) was granted by the European Commission to Omnicare Clinical Research, United Kingdom, for human Staphylococcus aureus polyclonal immunoglobulin and human Staphylococcus epidermidis polyclonal immunoglobulin for the prevention of late onset sepsis in premature infants of less than or equal to 32 weeks gestational age.

What is late onset sepsis in premature infants of less than or equal to 32 weeks gestational age?

Immunoglobulins are proteins produced by our body that help fighting infections. Newborns do not have the capacity to produce immunoglobulins themselves immediately after birth but have some immunoglobulins transferred from the mother during the last weeks of gestation. Premature babies, those born before the normal gestation duration (40 weeks), miss this period partially or completely and therefore they have a reduced amount of immunoglobulins or even lack them completely. For this reason they are more prone to suffer infections after birth as one of the natural defences of the body is not present. The risk of infection has been found to be particularly high for children born before or around 32 weeks of gestation. Late onset sepsis defines generalised infections that start 3 days after birth and in which the micro-organisms causing the infection can be identified from blood samples. Late onset sepsis in premature infants of less than 32 weeks gestational age is a life threatening condition as it increases the risk of death of premature babies.

What are the methods of prevention available?

Preventive measures against infections include adherence to the so-called universal precautions, such as limiting as much as possible any patient contact, using specialised hospital facilities for providing intensive nursing and medical care limited to premature babies (neonatal intensive care units), avoiding crowded intensive care units for premature babies, limiting the nurse-to-patient ratios, strict compliance with hand washing and very careful skin care of the premature babies. There are at present no products authorised for prevention of the condition.

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

Based on the information provided by the sponsor and previous knowledge of the Committee, the number of patients at risk of developing late onset sepsis in premature infants of less than or equal to 32 weeks gestational age was considered to be approximately 1.5 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 69,000 persons.

* Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union (EU 25), Norway, Iceland and Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

How is this medicinal product expected to act?

Staphylococcus aureus and Staphylococcus epidermidis are micro-organisms that are a normal component of the human flora, which are the microorganisms that normally live in areas of our body. However sometimes these micro-organisms can cause infections in human beings, particularly in patients with a compromised defence system (immunity) such as premature infants. Infections by Staphylococcus aureus and Staphylococcus epidermidis are amongst the most common causes of late onset sepsis in premature infants of less than 32 weeks gestational age. Human Staphylococcus aureus polyclonal immunoglobulin and human Staphylococcus epidermidis polyclonal immunoglobulin are immunoglobulins obtained form persons that have in their blood a very high content of immunoglobulin against both microorganisms. The administration of these immunoglobulins in premature babies would restore their defence system against these microorganisms. Therefore by replacing the missing immunoglobulins the product might protect the newborns against those infections.

What is the stage of development of this medicinal product?

At the time of submission of the application for orphan designation, clinical trials in premature babies of less than 32 weeks at risk of developing late onset sepsis were ongoing.
In the United States Orphan Drug status was granted for the reduction (prevention) of nosocomial bacteremia caused by Staphylococci in very low birth weight infants.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 21 October 2005 a positive opinion recommending the grant of the above-mentioned designation.

Opinions on orphan medicinal products designations are based on the following cumulative criteria:

• the seriousness of the condition,
• the existence or not of alternative methods of diagnosis, prevention or treatment and
• either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments.

Designated orphan medicinal products are still investigational products which were considered for designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of the quality, safety and efficacy will be necessary before this product can be granted a marketing authorisation.

EU/3/05/335: Public summary of positive opinion for orphan designation of human Staphylococcus aureus polyclonal immunoglobulin and human Staphylococcus epidermidis polyclonal immunoglobulin for the prevention of late on...

Adopted Reference Number: EMEA/COMP/367733/2005 Rev.1

Key facts

• Active substance:
  • Human Staphylococcus aureus immunoglobulin
  • human Staphylococcus epidermidis polyclonal immunoglobulin
• Intended use:
  Late onset sepsis in premature infants of less or equal than 32 weeks of gestational age
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/05/335
• Date of designation:
  14/12/2005
• Sponsor:

  Omnicare Clinical Research; Wessex Business Centre; Bumpers Way; Bumpers Farm; Chippenham; Wiltshire SN14 6NQ; United Kingdom; Telephone: [phone]; Telefax: [phone]