EU/3/17/1896

About

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
Sirolimus
Disease / condition
Treatment of pachyonychia congenita
Date of first decision
17/07/2017
Outcome
Positive
EU designation number
EU/3/17/1896

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 Esp S.L.
Ronda De Sant Pere 33 Ppal 3 Y 4
08010 Barcelona
Spain
Tel. +34 93 488 0972
E-mail: sabrina@raremoonconsulting.com

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.

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