Overview

On 14 December 2018, orphan designation (EU/3/18/2103) was granted by the European Commission to Albireo AB, Sweden, for (2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro-1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid (also known as A4250) for the treatment of biliary atresia.

Biliary atresia is a condition in which the bile ducts that transport bile from the liver towards the intestines are blocked or absent. As a result, the bile – which is used to digest fats – stays in the liver where it accumulates and causes damage. Symptoms of the disease appear a few weeks after birth.

Biliary atresia is a long-term 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 0.15 in 10,000 people in the European Union (EU). This was equivalent to a total of around 8,000 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).

*Disclaimer: 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 517,400,000 (Eurostat 2018).

At the time of application for orphan designation, there was no medicine authorised for biliary atresia. Patients had surgery to restore flow of the bile, and liver transplantation. They also received antibiotics, medicines to encourage bile flow, vitamin supplements and nutritional support.

The medicine blocks certain channels called IBATs through which the bile acids (a major component of bile) leave the intestines to reach the blood vessels that carry them back to the liver. By blocking these channels, the medicine is expected to reduce the amount of bile acids in the liver, thereby reducing the liver damage.

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 in patients with biliary atresia had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for biliary atresia or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 November 2018 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.

EU/3/18/2103: Public summary of opinion on orphan designation: (2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro-1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid

AdoptedReference Number: EMA/830039/2018

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Key facts

Intended use
Treatment of biliary atresia
Orphan designation status
Positive
EU designation number
EU/3/18/2103
Date of designation
Sponsor

Albireo AB
Arvid Wallgrens Backe 20
S-413 46 Gothenburg
Sweden
Tel. +46 31 741 14 80
E-mail: info@albireopharma.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:

  • 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:

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