EU/3/19/2213: Orphan designation for the treatment of growth hormone deficiency



On 17 October 2019, orphan designation EU/3/19/2213 was granted by the European Commission to Ascendis Pharma Endocrinology Division A/S, Denmark, for lonapegsomatropin for the treatment of growth hormone deficiency.

The sponsor’s address was updated in April 2021.

The medicinal product has been authorised in the EU as Lonapegsomatropin Ascendis Pharma since 11 January 2022.

Key facts

Active substance
Intended use
Treatment of growth hormone deficiency
Orphan designation status
EU designation number
Date of designation

Ascendis Pharma Endocrinology Division A/S
Tuborg Boulevard 12
2900 Hellerup 
Tel: +45 70 22 22 44

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of product at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.

More information is available in the PDF icon orphan medicine assessment report .

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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