EU/3/20/2358: Orphan designation for the treatment of amyotrophic lateral sclerosis

L-pyroglutamyl-L-asparaginyl-L-prolyl-D-tyrosyl-D-tryptophan amide

Table of contents


On 13 November 2020, orphan designation EU/3/20/2358 was granted by the European Commission to Neuropath Therapeutics Limited, Ireland, for L-pyroglutamyl-L-asparaginyl-L-prolyl-D-tyrosyl-D-tryptophan amide (also known as JAK4D) for the treatment of amyotrophic lateral sclerosis.

Key facts

Active substance
L-pyroglutamyl-L-asparaginyl-L-prolyl-D-tyrosyl-D-tryptophan amide
Intended use
Treatment of amyotrophic lateral sclerosis
Orphan designation status
EU designation number
Date of designation

Neuropath Therapeutics Limited
Suite 2006
26 Pembroke Street Upper
Dublin 2 DO2 X361
Tel: +353862240174

Documents related to this orphan designation evaluation

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:

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