EU/3/01/054 - orphan designation for treatment of meconium aspiration syndrome

sinapultide / Dipalmitoylphosphatidylcholine / palmitoyloleoyl phosphatidylglycerol / palmitic acid

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in April 2010 on request of the sponsor.

On 19 September 2001, orphan designation (EU/3/01/054) was granted by the European Commission to Discovery Laboratories Inc., United Kingdom, for sinapultide, dipalmitoylphosphatidylcholine, palmitoyl-oleyl phosphatidylglycerol and palmitic acid (Surfaxin) for the treatment of meconium aspiration syndrome.

What is meconium aspiration syndrome?

Meconium aspiration syndrome is a condition that affects newborns in consequence of inhalation of meconium (baby's first feces, ordinarily passed after birth) during delivery. Affected newborns develop respiratory distress (increasing difficulty in breathing) soon after birth. In brief, tiny air sacs called alveoli are located at the tips of the lungs' smallest breathing tubes, called bronchi. The alveoli are responsible for passing oxygen into the blood. When meconium is aspirated in the lungs, oxygen cannot enter the alveoli, which means that oxygen is no longer getting into the blood. Because the lungs are inflamed and filled with fluid, it is increasingly difficult to take breaths. Furthermore, the inflammation in the lungs leads to scarring. The lungs eventually become stiff with scar tissue and breathing becomes very difficult. Meconium aspiration syndrome is potentially chronically debilitating and life threatening.

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

At the time of designation, meconium aspiration syndrome affected approximately 0.25 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 9,600 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 the knowledge of the Committee for Orphan Medicinal Products (COMP).

* Disclaimer: The number of patients affected by the condition is estimated and assessed for the purpose of the designation, for a European Community population of 385,000,000 (Eurostat 2002) and may differ from the true number of patients affected by the condition. This estimate is based on available information and calculations presented by the sponsor at the time of the application.

What treatments are available?

At the time of submission of the application for orphan drug designation, no authorised medicinal products were available in the European Union. However, several therapeutic methods are available such as mechanical breathing assistance and/or removal of meconium through suction of the area of the throat at the back of the mouth and the windpipe.

Satisfactory argumentation has been submitted by the sponsor to justify the assumption that sinapultide, dipalmitoylphosphatidylcholine, palmitoyl-oleyl phosphatidylglycerol and palmitic acid might be of significant benefit for the treatment of meconium aspiration syndrome. 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?

Lung surfactant is a protein and fat complex that coats and keeps open the alveoli of the lung, thus ensuring a stable surface for oxygen passage. Surfaxin is a synthetic lung surfactant and the sponsor's hypothesis is that it will ultimately help the patient to take breaths more easily.

What is the stage of development of this medicine?

The effects of sinapultide, dipalmitoylphosphatidylcholine, palmitoyl-oleyl phosphatidylglycerol and palmitic acid were evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with meconium aspiration syndrome were ongoing.

Sinapultide, dipalmitoylphosphatidylcholine, palmitoyl-oleyl phosphatidylglycerol and palmitic acid was not marketed anywhere worldwide for treatment of meconium aspiration syndrome, at the time of submission. Orphan designation of sinapultide, dipalmitoylphosphatidylcholine, palmitoyl-oleyl phosphatidylglycerol and palmitic acid was granted in United States for treatment of meconium aspiration syndrome in newborn infants, treatment of acute respiratory distress syndrome in adults and in premature infants.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 18 July 2001 recommending the granting of this designation.

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

• the seriousness of the condition,
• the existence of alternative methods of diagnosis, prevention or treatment and
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the European Union) 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/3/01/054: Public summary of positive opinion for orphan designation of sinapultide, dipalmitoylphosphatidylcholine, palmitoyl-oleyl phosphatidylglycerol and palmitic acid for the treatment of meconium aspiration syndr...

Adopted Reference Number: EMEA/COMP/257/04 Rev. 2

Key facts

• Active substance:
  • sinapultide
  • Dipalmitoylphosphatidylcholine
  • palmitoyloleoyl phosphatidylglycerol
  • palmitic acid
• Intended use:
  Treatment of meconium aspiration syndrome
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/01/054
• Date of designation:
  19/09/2001
• Sponsor:

  Discovery Laboratories, Inc; Fountain Court; 32 Frances Road; Windsor; Berkshire SL4 3AA; United Kingdom; Telephone: [phone]; Telefax: [phone]; E-mail: [e-mail]