EU/3/15/1520: 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 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 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 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 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 512,900,000 (Eurostat 2015).

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.

Cannabidiol is a substance that is extracted from the cannabis plant, Cannabis sativa but which is considered not to have effects on mood, mental activity or behaviour. Although the way this medicine works in perinatal asphyxia is not clearly understood, cannabidiol is thought to have a protective effect on nerve cells due to its expected anti-inflammatory properties and ability to counteract the effect of toxic products containing oxygen. This is expected to improve the symptoms of the condition.

The effects of cannabidiol have been evaluated in experimental models.

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

At the time of submission, cannabidiol, in combination with delta-9-tetrahydrocannabinol, was authorised in some EU countries for the treatment of spasticity in multiple sclerosis.

At the time of submission, cannabidiol was not authorised anywhere in the EU for perinatal asphyxia. Orphan designation of cannabidiol had been granted in the United States for the treatment of neonatal hypoxic ischemic encephalopathy (NHIE).

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 18 June 2015 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.