EU/3/15/1493 - orphan designation for treatment of perinatal asphyxia
allopurinol sodium
OrphanHuman
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Overview
On 21 May 2015, orphan designation (EU/3/15/1493) was granted by the European Commission to ACE Pharmaceuticals BV, the Netherlands, for allopurinol sodium 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, as up to a fifth of the babies with the condition will die within the first days after birth.
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 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 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.
Allopurinol has been used for many years for the treatment of gout. It works by blocking an enzyme called xanthine oxidase, which plays an important part in breaking down substances in the body called 'purines' into a waste product, uric acid, a process whereby certain toxic products called 'free radicals' are also formed. Although is it not well understood how the medicine works in perinatal asphyxia, it is thought to prevent the accumulation of excess free radicals in babies, which is expected to improve the symptoms of the condition.
The sponsor has provided non-clinical and clinical data from the published literature to support its application for orphan designation.
At the time of submission, a clinical trial with allopurinol sodium in patients with perinatal asphyxia was planned.
At the time of submission, allopurinol 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 16 April 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.
Key facts
- Active substance
- allopurinol sodium
- Intended use
- Treatment of perinatal asphyxia
- Orphan designation status
- Positive
- EU designation number
- EU/3/15/1493
- Date of designation
- Sponsor
ACE Pharmaceuticals BV
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: