On 22 August 2014, orphan designation (EU/3/14/1314) was granted by the European Commission to Cerenis Therapeutics Holding SA, France, for recombinant human apolipoprotein A-I in a complex with phospholipids for the treatment of ATP-binding cassette transporter A1 deficiency.
Recombinant human apolipoprotein A-I in a complex with phospholipids
Treatment of ATP-binding cassette transporter A1 deficiency
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Review of designation
Documents related to this orphan designation evaluation
EU/3/14/1314: Public summary of opinion on orphan designation: Recombinant human apolipoprotein A-I in a complex with phospholipids for the treatment of ATP-binding cassette transporter A1 deficiency (PDF/123.6 KB)
First published: 30/09/2014
Last updated: 30/09/2014
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
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: