EU/3/04/217

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Orphan designation

On 29 July 2004, orphan designation (EU/3/04/217) was granted by the European Commission to GMG BioBusiness Ltd., United Kingdom, for Sinapultide, dipalmitoylphosphatidylcholine, palmitoyl-oleoyl phosphatidylglycerol and palmitic acid (Surfaxin) for the treatment of respiratory distress syndrome in premature neonates of less than 37 weeks of gestational age.
The sponsorship was transferred to Pharm Research Associates (UK) Limited, United Kingdom, in May 2009.

What is respiratory distress syndrome?

Respiratory distress syndrome (RDS) is a lung disorder that causes increasing difficulty in breathing. It may occur in adults (adult respiratory distress syndrome) or in newborn children, when it is also known as hyaline membrane disease. RDS in premature, low birth weight infants is characterised by the onset of difficulty in breathing within minutes to a few hours after birth. The condition is associated with the presence of deposits of a dense membrane (called hyaline membrane) in the air sacs (alveoli) of the lung. RDS is a life-threatening condition.

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

At the time of designation, respiratory distress syndrome affected approximately 1.5 in 10,000 premature neonates of less than 37 weeks of gestational age in the European Union (EU)*. This is equivalent to a total of around 69,000 people, 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 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 25), Norway, Iceland and Liechtenstein. This represents a population of 459,700,000 (Eurostat 2004).

What treatments are available?

Several surfactant products (see definition below) were authorised for the condition in the Community at the time of submission of the application for orphan drug designation. Other methods of treatment include the use of mechanical ventilatory support for the infant.
Satisfactory argumentation has been submitted by the sponsor to justify the assumption that Surfaxin might be of potential significant benefit for the treatment of RDS in premature neonates. The assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

Surfaxin is a surfactant agent. Lung surfactant is a protein and fat complex that coat the alveoli of the lung, and keep them open. This ensures a stable alveoli surface for oxygen passage.
Surfactant substances are naturally produced in lungs. In premature neonates the lungs have not fully matured and there may be a deficiency of natural surfactant. Surfaxin is intended to compensate for the lack of natural surfactant in infants with RDS and thus aims to improve the lung function.

What is the stage of development of this medicine?

 

The effects of sinapultide, dipalmitoylphosphatidylcholine, palmitoyl-oleoyl phosphatidylglycerol and palmitic acid were evaluated in experimental models. At the time of submission of the application for orphan designation, clinical trials in pemature neonates with RDS were completed.
This medicinal product was not marketed anywhere worldwide for RDS at the time of submission. Orphan designation of Surfaxin has previously been granted in the European Union for treatment of meconium aspiration syndrome (EU/3/01/054) and treatment of acute lung injury (EU/3/01/079).
In the United States orphan drug status has been granted for: treatment of acute respiratory distress syndrome in adults, treatment of respiratory distress syndrome in premature infants, and treatment of meconium aspiration syndrome in newborn infants.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 16 June 2004 a positive opinion recommending the grant of the above-mentioned 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 Community) 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>Sinapultide, dipalmitoylphosphatidylcholine, palmitoyloleoy phosphatidylglycerol and palmitic acid</p>
Active substanceSinapultide, dipalmitoylphosphatidylcholine, palmitoyloleoy phosphatidylglycerol and palmitic acid
Medicine Name
Disease/conditionRespiratory distress syndrome in premature neonates of less then 37 weeks of gestational age
Date of decision29/07/2004
OutcomePositive
Orphan decision numberEU/3/04/217

Review of designation

Sponsor’s contact details:

Pharm Research Associates (UK) Limited
Pacific House
Imperial Way
Reading, Berkshire RG2 0TD
United Kingdom
Telephone: +44 118 918 1000
Telefax: +44 118 918 1001

Patients association contact point:

BLISS (The premature baby charity)
68 South Lambeth Road
London SW8 1RL 
Telephone: +44 20 78 20 94 71
Parent Support Helpline FREEPHONE for UK citizens 0500 618140
Telefax: +44 20 78 20 95 67 
E-mail: information@bliss.org.uk