EU/3/18/2299: Orphan designation for the treatment of achondroplasia

C-type natriuretic peptide conjugated to multi-arm polyethylene glycol carrier through a cleavable linker

Overview

On 27 July 2020, orphan designation EU/3/20/2299 was granted by the European Commission to Ascendis Pharma Growth Disorders A/S, Denmark, for c-type natriuretic peptide conjugated to multi-arm polyethylene glycol carrier through a cleavable linker for the treatment of achondroplasia.

Key facts

Active substance
C-type natriuretic peptide conjugated to multi-arm polyethylene glycol carrier through a cleavable linker
Intended use
Treatment of achondroplasia
Orphan designation status
Positive
EU designation number
EU/3/18/2299
Date of designation
27/07/2020
Sponsor

Ascendis Pharma Endocrinology Division A/S
Tuborg Boulevard 12
2900 Hellerup 
Hovedstaden
Denmark
Tel: +45 70 22 22 44
E-mail: info@ascendispharma.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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