EU/3/16/1742: Orphan designation for the treatment of Smith-Magenis syndrome

Acebutolol hydrochloride

Table of contents

Overview

On 14 October 2016, orphan designation (EU/3/16/1742) was granted by the European Commission to Therapicon Srl, Italy, for acebutolol hydrochloride for the treatment of Smith-Magenis syndrome.

The sponsorship was transferred to Worphmed S.r.l., Italy in March 2019.

Key facts

Active substance
Acebutolol hydrochloride
Intended use
Treatment of Smith-Magenis syndrome
Orphan designation status
Positive
EU designation number
EU/3/16/1742
Date of designation
14/10/2016
Sponsor

Worphmed S.r.l.
Via Malachia Marchesi de Taddei 21
20146 Milan
Italy
Tel. +39 (02) 433042
E-mail: rare@worphmed.eu

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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