EU/3/20/2339: Orphan designation for the treatment of activated phosphoinositide 3-kinase delta syndrome

Leniolisib

Overview

On 19 October 2020, orphan designation EU/3/20/2339 was granted by the European Commission to Pharming Group N.V., the Netherlands, for leniolisib for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS).

The sponsorship was transferred to Pharming Technologies B.V., Netherlands in April 2022.

Key facts

Active substance
Leniolisib
Intended use
Treatment of activated phosphoinositide 3-kinase delta syndrome
Orphan designation status
Positive
EU designation number
EU/3/20/2339
Date of designation
19/10/2020
Sponsor

Pharming Technologies B.V.
Darwinweg 24
2333 CR Leiden
Zuid-Holland
Netherlands
E-mail: info@pharming.com

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Documents related to this orphan designation evaluation

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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