On 5 December 2016, orphan designation (EU/3/01/056) was granted by the European Commission to Genzyme Europe BV, the Netherlands, for recombinant human acid sphingomyelinase (also known as olipudase alfa) for the treatment of Niemann-Pick disease.
The medicinal product (for orphan indication in case of several orphan indications or type II variation) has been authorised in the EU as Xenpozyme since 24 June 2022.
Recombinant human acid sphingomyelinase (also known as olipudase alfa)
Treatment of Niemann-Pick disease
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Review of designation
The Committee for Orphan Medicinal Products reviewed the orphan designation of Xenpozyme at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.
More information is available in the orphan medicine assessment report.
|November 2018||The sponsor's address was updated in November 2018.|
Documents related to this orphan designation evaluation
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: