EU/3/20/2281: Orphan designation for the treatment of paroxysmal nocturnal haemoglobinuria

(4-{(2S,4S)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4-yl)methyl]piperidin-2-yl}benzoic acid-hydrogen chloride(1/1))


On 4 June 2020, orphan designation EU/3/20/2281 was granted by the European Commission to Novartis Europharm Limited, Ireland, for (4-{(2S,4S)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4-yl)methyl]piperidin-2-yl}benzoic acid-hydrogen chloride(1/1)) (also known as LNP023) for the treatment of paroxysmal nocturnal haemoglobinuria.

Key facts

Active substance
(4-{(2S,4S)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4-yl)methyl]piperidin-2-yl}benzoic acid-hydrogen chloride(1/1))
Intended use
Treatment of paroxysmal nocturnal haemoglobinuria
Orphan designation status
EU designation number
Date of designation

Novartis Europharm Limited
Vista Building
Elm Park, Merrion Road
Dublin 4
Tel.: +41 (0)61 324 11 11

Review of designation

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

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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