EU/3/12/982

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Orphan designation

On 2 April 2012, orphan designation (EU/3/12/982) was granted by the European Commission to Chiesi Farmaceutici S.P.A., Italy, for dipalmitoylphosphatidylcholine, 1-palmitoyl-2-oleoyl-sn-glycero-3-phosphoglycerol, sodium salt, synthetic surfactant protein C analogue and synthetic surfactant protein B analogue, for the treatment of respiratory distress syndrome in premature neonates of less than 37 weeks of gestational age.

What is respiratory distress syndrome in premature neonates?

Respiratory distress syndrome in premature neonates (newborn babies) is a breathing disorder in which the baby’s immature lungs are not producing enough surfactant. Surfactant is a substance lining the airways that allows the lungs to expand properly and is required in sufficient amounts for the newborn to start breathing after birth. Premature neonates with respiratory distress syndrome have problems breathing and require extra oxygen within minutes of being born.

Respiratory distress syndrome in premature neonates of less than 37 weeks of gestational age is a long-term and life-threatening condition because of the damage to the brain due to the lack of oxygen, and because of other complications resulting from treatment and mechanical ventilation (using a machine to help them to breathe).

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

At the time of designation, respiratory distress syndrome in premature neonates of less than 37 weeks gestational age affected approximately 2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 101,000 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 27), Norway, Iceland and Liechtenstein. This represents a population of 506,300,000 (Eurostat 2011).

What treatments are available?

At the time of designation, several surfactant products were authorised for the condition in the EU. Other methods of treatment included the use of mechanical ventilation.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for premature neonates with respiratory distress syndrome because early studies in experimental models indicate that it may act for longer than other surfactants and may improve the treatment of patients with 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?

This medicine is a synthetic surfactant designed to be very similar to natural surfactant. The medicine is expected to work by replacing the missing surfactant in the lungs of premature infants with respiratory distress syndrome. This will improve the child's lung function by keeping the airways open and allowing sufficient oxygen to be taken up.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.

At the time of submission, no clinical trials with the medicine in premature neonates with respiratory distress syndrome had been started.

At the time of designation, the medicine was not authorised anywhere in the EU for respiratory distress syndrome in premature neonates. Orphan designation of this medicine had been granted in the United States of America for the treatment of preterm neonatal respiratory distress syndrome.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 February 2012 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>Dipalmitoylphosphatidylcholine, 1-palmitoyl-2-oleoyl-sn-glycero-3-phosphoglycerol, sodium salt, synthetic surfactant protein C analogue and synthetic surfactant protein B analogue</p>
Active substanceDipalmitoylphosphatidylcholine, 1-palmitoyl-2-oleoyl-sn-glycero-3-phosphoglycerol, sodium salt, synthetic surfactant protein C analogue and synthetic surfactant protein B analogue
Medicine Name
Disease/conditionRespiratory distress syndrome in premature neonates of less than 37 weeks of gestational age
Date of decision02/04/2012
OutcomePositive
Orphan decision numberEU/3/12/982

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:

Chiesi Farmaceutici S.P.A.
Via Palermo 26/A
43122 Parma
Italy
Telephone: +39 0521 279 793
Telefax: +39 0521 77 41 20
info@chiesigroup.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.