EU/3/15/1483

On 24 April 2015, orphan designation (EU/3/15/1483) was granted by the European Commission to Neuroprotexeon Ltd, United Kingdom, for xenon for the treatment of perinatal asphyxia.

What is perinatal asphyxia?

Perinatal asphyxia happens when babies are born without enough oxygen in their blood. This is generally due to interruptions of the oxygen supplied by the mother through the placenta or the umbilical cord. Perinatal asphyxia can cause damage to the brain and other organs.

Perinatal asphyxia is a long-term debilitating disease because it can lead to the child being severely mentally and physically disabled. It is also life threatening, with up to a fifth of the babies with the condition dying within the first days after birth.

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

At the time of designation, perinatal asphyxia affected less than 1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 51,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

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*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 28), Norway, Iceland and Liechtenstein. This represents a population of 512,900,000 (Eurostat 2015).

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 they were sometimes cooled down to a body temperature lower than normal (hypothermia) for 12 to 72 hours after birth to reduce the extent of the damage caused by the asphyxia.

How is this medicine expected to work?

Xenon is a type of gas found in the atmosphere in very small amounts. Xenon has been investigated as a general anaesthetic, but it is also known to have a neuroprotective effect: this means that it can help protect nerve cells from the consequences of damage that occurs, for example, when nerve cells are starved of oxygen. Xenon is thought to attach to proteins known as NMDA receptors, which are found in nerve cells. NMDA receptors have been found to be overactivated in many pathological processes, including nerve cell death. By attaching to the receptors and blocking their action, xenon is expected to provide some protection against the damage caused by perinatal asphyxia.

What is the stage of development of this medicine?

The effects of xenon have been evaluated in experimental models.

At the time of submission of the application for orphan designation, some clinical trials with xenon in patients with perinatal asphyxia had concluded and further studies were planned.

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

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 19 March 2015 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.

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