EU/3/20/2312 - orphan designation for treatment of acute liver failure
allogeneic hepatoblastoma cells encapsulated in alginate, ex vivo expanded
OrphanHuman
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Overview
On 21 August 2020, orphan designation EU/3/20/2312 was granted by the European Commission to ESPL Regulatory Consulting Limited, Ireland, for allogeneic hepatoblastoma cells encapsulated in alginate, ex vivo expanded for the treatment of acute liver failure.
The sponsorship was transferred to UCL Research Limited, Ireland in June 2021.
Acute liver failure is the sudden loss of normal liver function in a patient with a previously normal liver and without evidence of chronic (long-term) liver disease.
The most common first sign of liver failure is jaundice (yellowing of the skin). Acute liver failure brings serious complications such as excessive bleeding due to impaired blood clotting, swelling around the brain, convulsions (fits) and coma.
The most common causes of acute liver failure are viral hepatitis (an infectious disease that affects the liver), overdose of medicines such as paracetamol or consumption of toxic substances such as poisonous mushrooms.
Acute liver failure is a life-threatening disease because of its damaging effects on the liver, brain and other organs.
At the time of designation, acute liver failure affected approximately 0.1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 5,000 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).
*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, the main treatment option for acute liver failure was liver transplantation. In addition, medicines were used to treat complications of liver failure such as hepatic encephalopathy. Early treatment with acetylcysteine was used to prevent liver failure due to paracetamol overdose.
The sponsor has provided sufficient information to show that this product might be of significant benefit for patients with acute liver failure because laboratory data suggest it could improve liver function. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The medicine is made of liver cells that have been modified and grown in a laboratory. The cells are embedded in a device to create a temporary artificial liver while the patient’s liver heals. It is expected that when plasma from the patient’s blood passes through this artificial liver, the liver cells in it will remove harmful substances, produce necessary new proteins and help to keep the correct balance of substances in the plasma. This can help to prevent some complications of acute liver failure.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with the medicine had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for the treatment of acute liver failure 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 16 July 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
- allogeneic hepatoblastoma cells encapsulated in alginate, ex vivo expanded
- Intended use
- Treatment of acute liver failure
- Orphan designation status
- Positive
- EU designation number
- EU/3/20/2312
- Date of designation
- Sponsor
UCL Research Limited
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: