On 22 May 2017, orphan designation (EU/3/17/1876) was granted by the European Commission to TMC Pharma Services Ltd, United Kingdom, for sodium (1R,3R,4R,5S)-3-({2-N-acetylamino-2-deoxy-3-O-[(1S)-1-carboxylato-2-cyclohexylethyl]-beta-D-galactopyranosyl}oxy)-4-({6-deoxy-alpha-L-galactopyranosyl}oxy)-5-ethyl-cyclohexan-1-yl-(38-oxo-2,5,8,11,14,17,20,23,26,29,32,35-dodecaoxa-39-azahentetracontan-41-yl)carboxamide (also known as GMI-1271) for the treatment of acute myeloid leukaemia.

The sponsorship was transferred to FGK Representative Service GmbH, Germany in March 2018.

Key facts

Active substance
Sodium (1R,3R,4R,5S)-3-({2-N-acetylamino-2-deoxy-3-O-[(1S)-1-carboxylato-2-cyclohexylethyl]-beta-D-galactopyranosyl}oxy)-4-({6-deoxy-alpha-L-galactopyranosyl}oxy)-5-ethyl-cyclohexan-1-yl-(38-oxo-2,5,8,11,14,17,20,23,26,29,32,35-dodecaoxa-39-azahentetracontan-41-yl)carboxamide
Disease / condition
Treatment of acute myeloid leukaemia
Date of decision
Orphan decision number

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

FGK Representative Service GmbH
Heimeranstrasse 35
80339 Munich

Patients' organisations

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.

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