Bylvay - withdrawal of application for variation to marketing authorisation

Application withdrawn

The application for a change to this medicine's authorisation has been withdrawn.

odevixibat

Overview

Albireo withdrew its application for the use of Bylvay to treat cholestatic pruritus (intense itching due to a build-up of bile in body tissues) in patients with Alagille syndrome (ALGS).

The company withdrew the application on 22 October 2023.

What is Bylvay and what is it used for?

Bylvay is a medicine for treating patients from the age of 6 months with progressive familial intrahepatic cholestasis (PFIC), a rare type of liver disease in which bile acids build up in the liver.

Bylvay has been authorised in the EU since July 2021.

It contains the active substance odevixibat and is available as capsules.

What change had the company applied for?

The company applied to extend the use of Bylvay to treat intense itching in patients with Alagille syndrome aged 6 months or older. Alagille syndrome is an inherited disease in which bile cannot drain properly from the liver, resulting in a build-up of bile acids in the liver and blood.

Bylvay was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 17 July 2012 for PFIC and on 9 August 2012 for Alagille syndrome.

How does Bylvay work?

The active substance in Bylvay, odevixibat, blocks the actions of IBAT, a protein in the intestine that transports bile acid from the intestines into the liver. By blocking the actions of IBAT, odevixibat reduces the amount of bile acid that is transported from the intestines into the liver. This reduces the build-up of bile acids and prevents damage to the liver tissue.

Bylvay works in the same way in people with PFIC and Alagille syndrome.

What did the company present to support its application?

The company presented results from a main study comparing Bylvay with placebo (a dummy treatment) in 52 patients aged from 6 months to 15.5 years with Alagille syndrome. The study measured the improvements in itching symptoms measured using a standard scoring system.

How far into the evaluation was the application when it was withdrawn?

The European Medicines Agency had completed its evaluation when the application was withdrawn by the company.

What did the Agency recommend at that time?

Based on the review of the information and the company’s response to the Agency’s questions, at the time of the withdrawal, the Agency had recommended authorising the use of Bylvay for treating cholestatic pruritus in patients with Alagille syndrome.

What were the reasons given by the company for withdrawing the application?

The company withdrew the application after EMA’s orphan medicines committee (COMP) indicated that it was going to issue a recommendation not to maintain the orphan designation for Alagille syndrome.

However, Bylvay has an orphan designation for PFIC and under EU law a medicine cannot be authorised for an orphan condition and a non-orphan one at the same time. More information about why the company withdrew is available in the company's withdrawal letter.

There are no concerns about the balance of the benefits and risk of Bylvay in patients with Alagille syndrome. The company is working on an application for a separate marketing authorisation for this condition.

Does this withdrawal affect patients in clinical trials or compassionate use programmes?

The company informed the Agency that there are no consequences for patients in the ongoing clinical trial or compassionate use programme.

What is happening with Bylvay for the treatment of PFIC?

There are no consequences for the use of Bylvay to treat PFIC.

Questions and answers on the withdrawal of application to change the marketing authorisation for Bylvay (odevixibat)

Reference Number: EMA/492792/2023

Key facts

• Name of medicine:
  Bylvay
• EMA product number:
  EMEA/H/C/004691
• Active substance:
  Odevixibat
• International non-proprietary name (INN) or common name:
  odevixibat
• Therapeutic area (MeSH):
  Cholestasis, Intrahepatic
• Anatomical therapeutical chemical (ATC) code:
  A05AX
• Accelerated assessment:

  This medicine had an accelerated assessment. This means that it is a medicine of major interest for public health, so its timeframe for review was 150 evaluation days rather than 210. For more information, see Accelerated assessment.

• Exceptional circumstances:

  This medicine was authorised under exceptional circumstances, because the applicant was unable to provide comprehensive data on the efficacy and safety of the medicine under normal conditions of use. This can happen because the condition to be treated is rare or because collection of full information is not possible or is unethical. For more information, see Pre-authorisation guidance.

• Orphan:

  This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

• PRIME: priority medicine:

  This medicine was granted entry to the EMA Priority Medicines (PRIME) scheme during its development. PRIME is a scheme launched by EMA to enhance support for the development of medicines that target an unmet medical need. This voluntary scheme is based on enhanced interaction and early dialogue with developers of promising medicines, to optimise development plans and speed up evaluation so these medicines can reach patients earlier. For more information, see PRIME: priority medicines.

• Marketing authorisation holder:
  Ipsen Pharma
• Date of issue of marketing authorisation valid throughout the European Union:
  16/07/2021
• Date of withdrawal:
  22/10/2023

Documents

Withdrawal assessment report for Bylvay (II-11)

Reference Number: EMA/371338/2023

Withdrawal letter: Bylvay (II-11)