EU/3/20/2304 - orphan designation for treatment of biliary atresia
Maralixibat chloride
OrphanHuman
Overview
On 27 July 2020, orphan designation EU/3/20/2304 was granted by the European Commission to Granzer Regulatory Consulting & Services, Germany, for maralixibat chloride for the treatment of biliary atresia.
The sponsorship was transferred to Mirum Pharmaceuticals International B.V., Netherlands, in September 2021.
Biliary atresia is a condition in which the bile ducts that transport bile from the liver to the intestines are blocked or absent. As a result, the bile – which is used to digest fats – builds up in the liver and causes damage. Symptoms of the disease appear a few weeks after birth.
Biliary atresia is a debilitating and life-threatening disease because of the long-term damage to the liver, including loss of liver tissue and function, and cirrhosis (scarring of the liver).
At the time of designation, biliary atresia 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 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 application for orphan designation, no medicine was authorised for the treatment of patients with biliary atresia. Patients had surgery to restore flow of the bile, or required liver transplantation. They also received antibiotics, medicines to encourage bile flow, vitamin supplements and nutritional support.
The medicine is expected to block certain channels called ASBTs through which bile acids (a major component of bile) leave the intestines to travel in the blood back to the liver. When these channels are blocked, the bile acids are instead excreted from the body. In this way, the medicine is expected to reduce the amount of bile acids in the liver and so reduce liver damage.
The effects of maralixibat chloride have been evaluated in experimental models.
At the time of submission of the application for orphan designation, no clinical trials with maralixibat chloride in patients with biliary atresia had been started.
At the time of submission, maralixibat chloride was not authorised anywhere in the EU for the treatment of biliary atresia 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 18 June 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
- Maralixibat chloride
- Intended use
- Treatment of biliary atresia
- Orphan designation status
- Positive
- EU designation number
- EU/3/20/2304
- Date of designation
- Sponsor
Mirum Pharmaceuticals International B.V.
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: