EU/3/17/1930 - orphan designation for treatment of primary biliary cholangitis
Seladelpar
OrphanHuman
Overview
On 16 October 2017, orphan designation (EU/3/17/1930) was granted by the European Commission to Larode Ltd, United Kingdom, for seladelpar for the treatment of primary biliary cholangitis.
Primary biliary 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 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 women.
Primary biliary 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 biliary cholangitis affected approximately 3.9 in 10,000 people in the European Union (EU). This was equivalent to a total of around 201,000 people1, 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).
1Disclaimer: 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 515,700,000 (Eurostat 2017).
At the time of designation, obeticholic acid and ursodeoxycholic acid were authorised in the EU for the treatment of primary biliary cholangitis, with obeticholic acid being used in patients who do not respond or cannot take ursodeoxycholic acid. In advanced cases, the patient may need a liver transplant.
The sponsor has provided sufficient information to show that seladelpar might be of significant benefit for patients with primary biliary cholangitis. Preliminary data showed that it may improve the outcome of patients who do not respond well to ursodeoxycholic acid and its effects compare well with those of obeticholic acid. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is expected to work by attaching to and activating receptors (targets) called 'PPAR gamma receptors', which regulate bile flow. By activating PPARs, this medicine is expected to reduce bile flow, thereby reducing damage of liver tissue seen in primary biliary cholangitis.
The effects of seladelpar have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with seladelpar in patients with primary biliary cholangitis were ongoing.
At the time of submission, seladelpar was not authorised anywhere in the EU for primary biliary cholangitis. Orphan designation of seladelpar had been granted in the United States for this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 September 2017 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
- Seladelpar
- Intended use
- Treatment of primary biliary cholangitis
- Orphan designation status
- Positive
- EU designation number
- EU/3/17/1930
- Date of designation
- Sponsor
Gilead Sciences Ireland UC
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 |
|---|---|
| February 2025 | The sponsorship was transferred to Gilead Sciences Ireland UC from Cymabay Ireland Limited. |
| January 2019 | The sponsorship was transferred to Cymabay Ireland Limited, Ireland. |
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: