EU/3/18/2031: Orphan designation for the treatment of perinatal asphyxia

Perinatal asphyxia happens when babies are born without enough oxygen in their blood. This is generally due to interruption of the oxygen supplied by the mother 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 days after birth.

At the time of designation, perinatal asphyxia affected approximately 0.4 in 10,000 people in the European Union (EU). This was equivalent to a total of around 21,000 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 28), Norway, Iceland and Liechtenstein. This represents a population of 517,400,000 (Eurostat 2018).

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 (therapeutic hypothermia) for 12 to 72 hours after birth to reduce the brain damage caused by the asphyxia.

Argon is a gas present in the atmosphere. The way argon works in babies with perinatal asphyxia is not fully understood. It is thought to reduce cell death and inflammation while activating processes that repair damaged cells. In addition, laboratory studies show that argon can protect nerve cells from the damage caused by low levels of oxygen.

The effects of argon have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with argon in patients with perinatal asphyxia had been started.

At the time of submission, argon was not authorised anywhere in the EU for perinatal asphyxia or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 24 May 2018 recommending the granting of this designation.

• 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.