EU/3/18/2010

Table of contents

About

On 16 April 2018, orphan designation (EU/3/18/2010) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for branaplam for the treatment of spinal muscular atrophy.

The sponsorship was transferred to Novartis Europharm Limited, Ireland, in September 2018.

Key facts

Active substance
branaplam
Disease / condition
Treatment of spinal muscular atrophy
Date of decision
16/04/2018
Outcome
Positive
Orphan decision number
EU/3/18/2010

Sponsor's contact details

Novartis Europharm Limited
Vista Building
Elm Park, Merrion Road
Dublin 4
Ireland
Tel.: +353 12204781
E-mail: orphan.enquiries@novartis.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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