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.
polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase (pegtibatinase)
Treatment of homocystinuria
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Review of designation
|February 2023||The sponsor’s address was updated.|
Documents related to this orphan designation evaluation
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: