EU/3/17/1896: Orphan designation for the treatment of pachyonychia congenita



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.

The sponsorship was transferred to Raremoon Consulting Esp S.L., Spain, in May 2020.

Key facts

Active substance
Intended use
Treatment of pachyonychia congenita
Orphan designation status
EU designation number
Date of designation

Raremoon Consulting Esp S.L.
Ronda De Sant Pere 33 Ppal 3 Y 4
08010 Barcelona
Tel. +34 93 488 0972

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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