EU/3/21/2515 - orphan designation for treatment of primary biliary cholangitis

Linerixibat

Overview

This medicine was designated as an orphan medicine for the treatment of primary biliary cholangitis in the European Union on 12 November 2021.

This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.

Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.

During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:

• EU Clinical Trials Register

• ClinicalTrials.gov

How the medicine is expected to work

Linerixibat works by reducing substances such as bile acids (a major component of bile) in the body that may cause cholestatic pruritus (itch) in primary biliary cholangitis (PBC). The itch can be intense, can occur throughout the body and is rarely relieved by scratching. Linerixibat is a tablet taken by mouth that acts locally in the intestine by blocking the ileal bile acid transport protein (IBAT). This reduces the return of bile acids through the blood to the liver and removes excess bile acids through the stool.

Stage of development at time of orphan designation

At the time of submission of the application for orphan designation: 

• The effects of the medicine had been evaluated in experimental models. 
• Clinical trials with the medicine in patients with primary biliary cholangitis were ongoing. 

More information on how potential new medicines are tested during their development is available on Authorisation of medicines.

About orphan designation

Medicines intended for rare diseases can be granted an orphan designation during their development.

The orphan designation allows the developer to benefit from:

• scientific and regulatory support to advance their medicine to the stage where they can request marketing authorisation;
• market exclusivity once the medicine is on the market.

To qualify for orphan designation, a medicine must meet a number of criteria:

• it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
• the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;
• there are no satisfactory alternative methods for the diagnosis, prevention or treatment of the condition or, if such a method exists, the medicine is of significant benefit to those affected by the condition.

EMA's Committee for Orphan Medicinal Products (COMP) is responsible for issuing opinions on applications for orphan designations.

The Agency sends the COMP opinion to the European Commission, which is responsible for granting the orphan designation. The full list of orphan designations is available in the Community register of orphan medicinal products for human use.

For more information, see:

• Orphan designation: Overview

• Rare diseases, orphan medicines - Getting the facts straight

Minutes of the COMP meeting 5-7 October 2021

Adopted

Key facts

• Active substance:
  Linerixibat
• Intended use:
  Treatment of primary biliary cholangitis
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/21/2515
• Date of designation:
  12/11/2021
• Sponsor:

  GlaxoSmithKline (Ireland) Limited; 12 Riverwalk; Citywest Business Campus; Dublin 24; D24 YK11; Ireland; E-mail: [e-mail]