EU/3/17/1873

About

On 22 May 2017, orphan designation (EU/3/17/1873) was granted by the European Commission to Best Regulatory Consulting Ltd, United Kingdom, for poly(oxy-1,2-ethanediyl),.alpha.-hydro-.omega.-hydroxy-,15,15'-diester with N-acetyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-.alpha.-aspartyl-L-tryptophylglycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-L-threonyl-2-[2-(2-aminoethoxy)ethoxy]acetyl-N6-carboxy-L-lysinamide cyclic (2.fwdarw.12)-(disulfide); where two identical synthetic peptide domains are covalently linked at the ends of the polyethylene glycol chain (also known as APL-2) for the treatment of paroxysmal nocturnal haemoglobinuria.

The sponsorship was transferred to Apellis Ireland Limited, Ireland, in April 2019.

Key facts

Active substance
Poly(oxy-1,2-ethanediyl),.alpha.-hydro-.omega.-hydroxy-,15,15'-diester with N-acetyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-.alpha.-aspartyl-L-tryptophylglycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-L-threonyl-2-[2-(2-aminoethoxy)ethoxy]acetyl-N6-carboxy-L-lysinamide cyclic (2.fwdarw.12)-(disulfide); where two identical synthetic peptide domains are covalently linked at the ends of the polyethylene glycol chain
Disease / condition
Treatment of paroxysmal nocturnal haemoglobinuria
Date of first decision
22/05/2017
Outcome
Positive
EU designation number
EU/3/17/1873

Review of designation

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

Sponsor's contact details

Apellis Ireland Limited
10 Earlsfort Terrace
Dublin 2 D02 T380
Ireland
Tel: + 353 (01) 920 1256
E-mail: davidw@apellis.com

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;

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