EU/3/17/1896:

Sirolimus

Overview

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
Intented use
Treatment of pachyonychia congenita
Date of designation
17/07/2017
Orphan designation status
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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