EU/3/20/2335: Orphan designation for the treatment of sickle cell disease



On 19 October 2020, orphan designation EU/3/20/2335 was granted by the European Commission to Clinipace GmbH, Germany, for (S)-1-(5-((2,3-dihydro-[1,4]dioxino[2,3-b]pyridin-7-yl)sulfonyl)-3,4,5,6-tetrahydropyrrolo[3,4-c]pyrrol-2(1H)-yl)-3-hydroxy-2-phenylpropan-1-one (also known as FT-4202) for the treatment of sickle cell disease.

Key facts

Active substance
Intended use
Treatment of sickle cell disease
Orphan designation status
EU designation number
Date of designation

Novo Nordisk A/S
Novo Alle 1
2880 Bagsvaerd

Review of designation

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

Update history

September 2023The sponsorship was transferred from Clinipace GmbH, Germany to Novo Nordisk A/S, Denmark.

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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