On 9 December 2011, orphan designation (EU/3/11/935) was granted by the European Commission to Prof. Roberto Testi, Italy, for interferon gamma for the treatment of Friedreich's ataxia.

The sponsorship was transferred to Horizon Pharma Ireland Limited, Ireland, in November 2016.

Key facts

Active substance
Interferon gamma
Disease / condition
Treatment of Friedreich's ataxia
Date of decision
Orphan decision number

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

Horizon Pharma Ireland Limited
Connaught House, 1st Floor
1 Burlington Road
Dublin 4
D04 C5Y6
Tel. +353 1 772 2137

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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