EU/3/16/1664: Orphan designation for the treatment of homocystinuria

polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase (pegtibatinase)

Overview

On 30 May 2016, orphan designation (EU/3/16/1664) was granted by the European Commission to Alan Boyd Consultants Ltd, United Kingdom, for polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase for the treatment of homocystinuria.

The sponsorship was transferred to Boyd Consultants Limited, Ireland, in March 2019.

The sponsor’s address was updated in September 2021.

The sponsorship was transferred to Travere Therapeutics Ireland Limited., Ireland, in April 2022.

Key facts

Active substance
polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase (pegtibatinase)
Intended use
Treatment of homocystinuria
Orphan designation status
Positive
EU designation number
EU/3/16/1664
Date of designation
30/05/2016
Sponsor

Travere Therapeutics Ireland Limited
2nd Floor
Pepper Canister House
3 Mount Street Crescent
Dublin 2
Co. Dublin
D02 WC63
Ireland
Email: info@travere.com

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

This medicine is now known as pegtibatinase.

Update history

DateUpdate
February 2023The sponsor’s address was updated.

Documents related to this orphan designation evaluation

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:

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