EU/3/12/1080: Orphan designation for the treatment of chronic lymphocytic leukaemia

4-(4-{[2-(4-Chlorophenyl)-4,4-dimethylcyclohex-1-en-1-yl]methyl}piperazin-1-yl)-N-({3-nitro-4-[(tetrahydro-2H-pyran-4-ylmethyl)amino]phenyl}sulfonyl)-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamide (venetoclax)


Please note that this product (marketed as Venclyxto) was withdrawn from the Community Register of designated orphan medicinal products in October 2018 upon request of the marketing authorisation holder at the time of the granting of a change to the terms of the marketing authorisation.

On 6 December 2012, orphan designation (EU/3/12/1080) was granted by the European Commission to AbbVie Ltd, United Kingdom, for 4-(4-{[2-(4-chlorophenyl)-4,4-dimethylcyclohex-1-en-1-yl]methyl}piperazin-1-yl)-N-({3-nitro-4-[(tetrahydro-2H-pyran-4-ylmethyl)amino]phenyl}sulfonyl)-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamide for the treatment of chronic lymphocytic leukaemia.

4-(4-{[2-(4-chlorophenyl)-4,4-dimethylcyclohex-1-en-1-yl]methyl}piperazin-1-yl)-N-({3-nitro-4-[(tetrahydro-2H-pyran-4-ylmethyl)amino]phenyl}sulfonyl)-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamide has been authorised in the EU as Venclyxto since 5 December 2016.

The sponsorship was transferred to AbbVie Deutschland GmbH & Co. KG, Germany, in March 2018.

This medicine is now known as venetoclax.

Key facts

Active substance
4-(4-{[2-(4-Chlorophenyl)-4,4-dimethylcyclohex-1-en-1-yl]methyl}piperazin-1-yl)-N-({3-nitro-4-[(tetrahydro-2H-pyran-4-ylmethyl)amino]phenyl}sulfonyl)-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamide (venetoclax)
Medicine name
Intended use
Treatment of chronic lymphocytic leukaemia
Orphan designation status
EU designation number
Date of designation

AbbVie Deutschland GmbH & Co. KG
67061 Ludwigshafen Am Rhein
Tel. +49 621 589 3382

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Venclyxto at the time of marketing authorisation.

The sponsor formally requested the withdrawal of the orphan designation prior to the Committee’s final opinion. More information is available in the PDF icon withdrawal assessment report – orphan maintenance .

On 14 October 2016, the Committee for Orphan Medicinal Products (COMP) completed the review of the designation EU/3/12/1080 for Venclyxto (venetoclax1) as an orphan medicinal product for the treatment of chronic lymphocytic leukaemia. 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 methods of treatment. As other methods of treatment are authorised in the European Union (EU), the COMP also considered whether the medicine is of significant benefit to patients with chronic lymphocytic leukaemia. The COMP recommended that the orphan designation of the medicine be maintained2.

1Previously known as 4-(4-{[2-(4-chlorophenyl)-4,4-dimethylcyclohex-1-en-1-yl]methyl}piperazin-1-yl)-N-({3-nitro-4-[(tetrahydro-2H-pyran-4-ylmethyl)amino]phenyl}sulfonyl)-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamide.

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 the same 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: Venclyxto (venetoclax) for the treatment of chronic lymphocytic leukaemia (PDF/94.62 KB)


    First published: 21/12/2016
    Last updated: 22/11/2018

  • 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:

    How useful was this page?

    Add your rating
    1 rating