EU/3/18/2115: Orphan designation for the treatment of spinal muscular atrophy

human anti-promyostatin monoclonal antibody (apitegromab)


This medicine is now known as apitegromab.

On 14 December 2018, orphan designation (EU/3/18/2115) was granted by the European Commission to Yes Pharmaceutical Development Services GmbH, Germany, for human anti-promyostatin monoclonal antibody (also known as SRK-015) for the treatment of spinal muscular atrophy.

The sponsorship was transferred to Diamond Pharma Services Ireland Limited, Ireland, in January 2021.

Key facts

Active substance
human anti-promyostatin monoclonal antibody (apitegromab)
Intended use
Treatment of spinal muscular atrophy
Orphan designation status
EU designation number
Date of designation

Propharma Group The Netherlands B.V.
Schipholweg 73
2316 ZL Leiden

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

Date Update
September 2022 The sponsorship was transferred to Propharma Group The Netherlands B.V., Netherlands in September 2022

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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