EU/3/19/2201

About

On 21 August 2019, orphan designation EU/3/19/2201 was granted by the European Commission to Apellis Ireland Limited, Ireland, 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 C3 glomerulopathy.

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 C3 glomerulopathy
Date of first decision
21/08/2019
Outcome
Positive
EU designation number
EU/3/19/2201

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
Co. Dublin
Ireland
Tel: +353 (1) 920 1256
E-mail: ireland@apellis.com

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

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