This medicine was designated as an orphan medicine for the treatment of hypertrophic cardiomyopathy due to mutations in the MYBPC3 gene encoding cardiac myosin-binding protein C in the European Union on 13 April 2022.
This means that the developer will receive scientific and regulatory support from EMA to advance their medicine to the stage where they can apply for a marketing authorisation.
Orphan designation does not mean the medicine is available or authorised for use. All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
During the medicine's development, doctors may be able to enrol patients in clinical trials investigating the medicine. For information on ongoing clinical trials in the EU, see:
Adeno-associated virus serotype 9 containing human MYBPC3 gene
Treatment of Hypertrophic cardiomyopathy due to mutations in the MYBPC3 gene encoding cardiac myosin-binding protein C
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Yes Pharmaceutical Development Services GmbH
Minutes of the COMP meeting on 15-17 March 2022 (PDF/505.22 KB)
First published: 13/06/2022
Last updated: 22/09/2022
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: