EU/3/20/2314 - orphan designation for treatment of primary sclerosing cholangitis
Humanised IgG1 monoclonal antibody against human eotaxin-2
nebokitug
Orphan
Human
Overview
On 21 August 2020, orphan designation EU/3/20/2314 was granted by the European Commission to Granzer Regulatory Consulting & Services, Germany, for humanised IgG1 monoclonal antibody against human eotaxin-2 (also known as CM-101) for the treatment of primary sclerosing cholangitis.
Primary sclerosing cholangitis is a disease in which there is long-term damage to the small bile ducts in the liver. These ducts transport fluid called bile from the liver towards the intestines, where it is used to help digest fats. Because of the damage to the ducts, bile acids, essential components of bile, build up in the liver causing inflammation and damage to liver tissue and leading to liver cirrhosis (scarring of the liver). Early symptoms of the disease include tiredness and itching. The disease is more common in middle-aged men.
Primary sclerosing cholangitis is a long-term debilitating and life-threatening disease because, when the disease progresses, it may lead to liver cirrhosis and liver failure, and may increase the risk of liver cancer.
At the time of designation, primary sclerosing cholangitis affected approximately 2.5 in 10,000 people in the European Union (EU). This was equivalent to a total of around 130,000 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, ursodeoxycholic acid was authorised in some EU countries for the treatment of primary sclerosing cholangitis. In advanced cases, the patient may need a liver transplant.
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with primary sclerosing cholangitis because it works in a different way to existing treatments and laboratory results suggest that unlike them it could reduce inflammation and scarring of the liver.
This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The medicine is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to a protein called eotaxin-2 (also called chemokine CCL24) which is involved in the inflammation process and the formation of fibrous tissue. By blocking eotaxin-2, this medicine is expected to reduce inflammation and scarring of the liver, thereby relieving the symptoms of the disease.
The effects of Humanised IgG1 monoclonal antibody against human eotaxin-2 have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with primary sclerosing cholangitis had started.
At the time of submission, the medicine was not authorised anywhere in the EU for the treatment of primary sclerosing cholangitis. Orphan designation of the medicine had been granted in the United Sates for the 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
- Humanised IgG1 monoclonal antibody against human eotaxin-2
- nebokitug
- Intended use
- Treatment of primary sclerosing cholangitis
- Orphan designation status
- Positive
- EU designation number
- EU/3/20/2314
- Date of designation
- Sponsor
FGK Representative Service GmbH
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Update history
| Date | Update |
|---|---|
| June 2025 | The sponsorship was transferred from Granzer Regulatory Consulting & Services to FGK Representative Service GmbH |
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: