EU/3/18/2127 - orphan designation for treatment of perinatal asphyxia

melatonin

Overview

On 11 January 2019, orphan designation (EU/3/18/2127) was granted by the European Commission to Therapicon S.r.l., Italy, for melatonin for the treatment of perinatal asphyxia.

The sponsorship was transferred to Worphmed S.r.l. Italy, in June 2019.

What is perinatal asphyxia?

Perinatal asphyxia happens when babies are born without enough oxygen in their blood. This is generally due to interruption in the supply of oxygen from the mother to the child through the umbilical cord. Perinatal asphyxia can cause damage to the brain and other organs.

Perinatal asphyxia is a long-term debilitating condition because it can lead to the child being mentally and physically disabled. It is also life threatening, with up to 1 baby in 5 with the condition dying within the first few days after birth.

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

At the time of designation, perinatal asphyxia affected approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 52,000 people¹, 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).

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¹Disclaimer: 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 28), Norway, Iceland and Liechtenstein. This represents a population of 517,400,000 (Eurostat 2018).

What treatments are available?

At the time of orphan designation, there was no treatment for perinatal asphyxia authorised in the EU. Babies with perinatal asphyxia received supportive treatment, and were sometimes cooled down to a body temperature lower than normal (therapeutic hypothermia) for 12 to 72 hours after birth to reduce the brain damage caused by the asphyxia.

How is this medicine expected to work?

Melatonin is a natural substance produced in the body with a variety of activities. The exact way it works in perinatal asphyxia is not fully understood, but data from experimental models show that it can reduce the production of damaging substances in the cell following changes in oxygen levels and has a protective effect on nerve cells, increasing their survival.

What is the stage of development of this medicine?

The effects of melatonin have been evaluated in experimental models.

At the time of submission, clinical trials with melatonin in patients with perinatal asphyxia had been carried out.

At the time of submission, melatonin was not authorised anywhere in the EU for treatment of perinatal asphyxia. Orphan designation of the medicine had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 December 2018 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.

EU/3/18/2127: Public summary of opinion on orphan designation: Melatonin for the treatment of perinatal asphyxia

Reference Number: EMADOC-628903358-228

Key facts

• Active substance:
  melatonin
• Intended use:
  Treatment of perinatal asphyxia
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/18/2127
• Date of designation:
  11/01/2019
• Sponsor:

  Worphmed S.r.l.

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.