On 17 July 2017, orphan designation (EU/3/17/1896) was granted by the European Commission to Raremoon Consulting Ltd, United Kingdom, for sirolimus (also known as PTX-001) for the treatment of pachyonychia congenita.

Key facts

Active substance
Disease / condition
Treatment of pachyonychia congenita
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

Raremoon Consulting Ltd
International House
24 Holborn Viaduct
City of London
London EC1A 2BN
United Kingdom
Tel. +44 203 5145553

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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