EU/3/07/518: Orphan designation for the treatment of pulmonary arterial hypertension

Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate (riociguat)  

Overview

On 20 December 2007, orphan designation (EU/3/07/518) was granted by the European Commission to Bayer HealthCare AG, Germany, for Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4 b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate for the treatment of pulmonary arterial hypertension including chronic thromboembolic pulmonary hypertension.

The sponsorship was transferred to Bayer Schering Pharma AG, Germany, in April 2009. Bayer Shering Pharma AG changed its name to Bayer Pharma AG in October 2011.

The sponsorship was transferred to Bayer AG, Germany, in August 2017.

Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate has been authorised in the EU as Adempas since 27 March 2014.

This medicine is now known as riociguat.

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in June 2022 upon request of the marketing authorisation holder.

Key facts

Active substance
Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate (riociguat)  
Medicine name
Adempas
Intended use
Treatment of pulmonary arterial hypertension
Orphan designation status
Withdrawn
EU designation number
EU/3/07/518
Date of designation
20/12/2007
Sponsor

Bayer AG
51368 Leverkusen
Germany
Tel. +49 30 300 139 003
E-mail: clinical-trials-contact@bayer.com

Review of designation

During its meeting of 4 to 6 February 2014, the Committee for Orphan Medicinal Products (COMP) reviewed the designation EU/3/07/518 for Adempas (riociguat1) as an orphan medicinal product for the treatment of pulmonary arterial hypertension including chronic thromboembolic pulmonary 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 maintained2.


1Previously known as methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4 b]pyridine-3-yl]-5-pyrimidinyl(methyl) carbamate.

2The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with a comparable therapeutic indication cannot be placed on the market.

Documents related to this orphan designation evaluation

  • List item

    Recommendation for maintenance of orphan designation at the time of marketing authorisation: Adempas (riociguat) for the treatment of pulmonary arterial hypertension including chronic thromboembolic pulmonary hypertensio... (PDF/144.2 KB)

    Adopted

    First published: 24/04/2014
    Last updated: 14/06/2022
    EMA/COMP/68649/2014

  • 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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