EU/3/20/2283 - orphan designation for treatment of neonatal encephalopathy
Ile-Ala-Leu-Ile-Leu-Glu-Pro-Ile-Cys-Cys-Gln-Glu-Arg-Ala-Ala-(discrete-polyethylene glycol)24
OrphanHuman
Overview
On 4 June 2020, orphan designation EU/3/20/2283 was granted by the European Commission to Clinipace GmbH, Germany, for Ile-Ala-Leu-Ile-Leu-Glu-Pro-Ile-Cys-Cys-Gln-Glu-Arg-Ala-Ala-(discrete-polyethylene glycol)24 (also known as PIC1-dPEG24) for the treatment of neonatal encephalopathy.
Neonatal encephalopathy refers to brain damage that occurs around the time of birth in babies who are not premature. Oxygen deprivation at birth is one of the most common causes of neonatal encephalopathy. Symptoms include reduced level of consciousness, seizures (fits), difficulty breathing, low muscle tone and poor reflexes.
Neonatal encephalopathy is a long-term debilitating disease due to its effects on mental and physical development, and can be life-threatening in the most severe cases.
At the time of designation, neonatal encephalopathy affected less than 1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 52,000 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).
*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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).
At the time of designation, babies with neonatal encephalopathy received therapeutic hypothermia, whereby the baby's body is cooled down to a body temperature lower than normal (hypothermia) to reduce extent of damage.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with neonatal encephalopathy because early data suggest that the medicine might benefit patients when used together with therapeutic hypothermia.
This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Low levels of oxygen in the brain can activate the immune system leading to inflammation and the production of harmful types of oxygen called reactive oxygen species, which can damage brain cells.
The medicine is expected to act on a protein called ‘C1q’, which is part of the body’s immune system. The medicine is also expected to block certain enzymes involved in the production of reactive oxygen species, thereby helping to prevent inflammation and damage and relieve symptoms of the condition.
At the time of submission of the application for orphan designation, the evaluation of the effects of the medicine in experimental models was ongoing.
At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with neonatal encephalopathy had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for the treatment of neonatal encephalopathy or designated as an orphan medicinal product elsewhere for this condition.
In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 23 April 2020, 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
- Ile-Ala-Leu-Ile-Leu-Glu-Pro-Ile-Cys-Cys-Gln-Glu-Arg-Ala-Ala-(discrete-polyethylene glycol)24
- Intended use
- Treatment of neonatal encephalopathy
- Orphan designation status
- Positive
- EU designation number
- EU/3/20/2283
- Date of designation
- Sponsor
Clinipace GmbH
Helfmann-Park 10
65760 Eschborn
Germany
Tel: +1 757 901 0460
E-mail: info@realtalifesciences.com
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: