European Medicines Agency - Human Medicines - Referrals - Article 29 paediatric referrals

Human Medicines – Referrals
List of Referrals – Article 29 – ‘paediatric referrals’
Background
Article 29 of Regulation (EC) No 1901/2006 as amended foresees that an application as referred to in article 8 of the said regulation can be submitted, in accordance with the procedure laid down in articles 32, 33 and 34 of Directive 2001/83/EC, for products authorised under Directive 2001/83/EC.

In this referral procedure, the marketing authorisation holders may apply for a new indication, a new pharmaceutical form or a new route of administration.

The application shall comply with the requirements laid down in point (a) of article 7(1) of Regulation (EC) No 1901/2006, regarding the inclusion of the results of all studies performed and details of all information collected in compliance with an agreed paediatric investigation plan.

At the end of the referral, the CHMP opinion may contain a recommendation for some changes to the prescribing information (such as the Summary of Product Characteristics wording), for the approval of a new pharmaceutical form and/or a new route of administration. It can also contain some conditions to the marketing authorisation, to ensure the safe and effective use of the medicine in the paediatric population. The assessment is limited to the specific sections of the product information to be varied.

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European Medicines Agency – Human Medicines – Referrals – Article 29 paediatric referrals

late-onset sepsis in premature infants of less than or equal to 32 weeks of gest - European Medicines Agency - Rare disease designations - EU/3/10/755

On 27 July 2010, orphan designation (EU/3/10/755) was granted by the European Commission to Schülke & Mayr GmbH, Germany, for octenidine dihydrochloride for the prevention of late-onset sepsis in premature infants of less than or equal to 32 weeks of gestational age.

What is late-onset sepsis?
Late-onset sepsis is a severe bacterial infection of the blood that occurs in newborn babies at least three days after birth. Premature babies born eight weeks or more too early (32 weeks of gestational age or less) have a higher risk of late-onset 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 by injection, which can put them at risk of infections in neonatal intensive care units.
Late-onset sepsis is a life-threatening condition that is one of the causes of high mortality 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 born at 32 weeks of gestational age or less was estimated to be approximately 1.5 people in 10,000 in the European Union (EU)*. This is equivalent to a total of around 76,000 babies, and is below the threshold 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. This represents a population of 506,500,000 (Eurostat 2010).

What methods of prevention are available?
At the time of designation, no satisfactory methods were authorised in the EU for the prevention of late-onset sepsis in these babies. Common infection-control measures such as hand-washing were used to keep the infection rate as low as possible in neonatal intensive care units. In some cases, antibiotics and skin antiseptics were also used to prevent infection.

How is this medicine expected to work?
Octenidine dihydrochloride is an antiseptic that has been available in the EU since 1997 in two medicines, which also contain alcohol, for disinfecting the skin and moist body surfaces in children and adults. Octenidine dihydrochloride works by blocking the growth of bacteria, fungi and other organisms that can cause infection.
This medicine is an aqueous solution of octenidine dihydrochloride. The absence of alcohol is expected to make it suitable for use on the sensitive skin of premature babies.

What is the stage of development of this medicine?
The effects of octenidine dihydrochloride have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with octenidine dihydrochloride in premature babies born at 32 weeks of gestational age or less had been started.
At the time of submission, octenidine dihydrochloride as single agent was not authorised anywhere in the EU for the prevention of late-onset sepsis in premature infants or designated as an orphan medicinal product elsewhere for this condition.
In accordance with Regulation (EC) No141/2000-of 16 December 1999, the COMP adopted a positive opinion on 8 April 2010 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteriathe 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.

EU/3/10/755: Public summary of opinion on orphan designation: Octenidine dihydrochloride for the prevention of late-onset sepsis in premature infants of less than or equal to 32 weeks of gestational age
http://www.ema.europa.eu/docs/en_GB/document_library/Orphan_designation/2010/08/WC500095702.pdf

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European Medicines Agency – Rare disease designations – EU/3/10/755