EU/3/16/1664: Orphan designation for the treatment of homocystinuria
polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase (pegtibatinase)
Table of contents
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 |
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
Date | Update |
---|---|
February 2023 | The sponsor’s address was updated. |
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: