EU/3/11/909: Orphan designation for the treatment of pulmonary arterial hypertension



On 27 September 2011, orphan designation (EU/3/11/909) was granted by the European Commission to Actelion Registration Limited, United Kingdom, for macitentan for the treatment of pulmonary arterial hypertension.

Macitentan has been authorised in the EU as Opsumit since 20 December 2013.

The sponsorship was transferred to Janssen-Cilag International N.V., Belgium, in September 2018.

Key facts

Active substance
Medicine name
Intended use
Treatment of pulmonary arterial hypertension
Orphan designation status
EU designation number
Date of designation

Janssen-Cilag International N.V.
Turnhoutseweg, 30
2340 Beerse
Tel. +32 146 031 89

Review of designation

During its meeting of 5-6 November 2013, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/11/909 for Opsumit (macitentan) as an orphan medicinal product for the treatment of pulmonary arterial hypertension. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other satisfactory methods of treatment. As other methods of treatment for patients with this condition are authorised in the European Union (EU), the COMP also looked at the significant benefit of the product over existing treatments. The COMP recommended that the orphan designation of the medicine be maintained.

Documents related to this orphan designation evaluation

  • List item

    Recommendation for maintenance of orphan designation at the time of marketing authorisation: Opsumit (macitentan) for the treatment of pulmonary arterial hypertension (PDF/79.42 KB)


    First published: 07/02/2014
    Last updated: 07/02/2014

  • 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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