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  • Orphan Drugs may be searched by the name of the substance in their composition, or by their commercial name (Trade name). Substance names include chemical names, code names of the substance or common names (INN : International Non-proprietary Name).
    You may use truncated names. Fill in the field “Substance/Trade name” and click OK. A list of all drugs matching your query will appear. Select the one of interest to you by clicking on it.


  • Orphan drugs may be searched by the name(s) of the disease(s) to which they are linked.
    This can be the disease(s) for which they have been granted an orphan designation (for more information on the orphan designation procedure, see List of Orphan Designations) or the disease(s) corresponding to the therapeutic indication when the drug has been granted a marketing authorisation.
    You may use truncated names. Fill in the field “name of disease” and click OK. A list of all diseases matching your query will appear. Select the one of interest to you by clicking on it.
    You can restrict your search to only the medical products that have been granted an orphan designation and/or a marketing authorisation, and/or to a specific geographical area.


  • Orphan drugs may be searched by ATC category. ATC categories correspond to the WHO’s Anatomical, Therapeutic, Chemical classification, which is used for the classification of medicinal substances.
    Select an ATC category and click OK.
    A list of all drugs matching your query will appear. Select the one of interest to you by clicking on it.
    You can restrict your search to only the medical products linked to a particular status (orphan designation, marketing authorisation,…) or to a specific geographical area.


  • Orphan drugs may be searched by the name of the sponsor of orphan designation when they have been granted an orphan designation and by the name of the MA holder when they have been granted a marketing authorisation. Sponsors or MA holders are usually pharmaceutical or biotech companies, but sometimes, institutions or natural persons.
    You may use truncated names. Fill in the field « Name of the sponsor / MA holder » and click OK.
    A list of all the names of sponsors of orphan designation or MA holders matching your query will appear. Select the one of interest to you by clicking on it.

The list of Orphan Drugs in the Orphanet database includes all the substances which have been granted an orphan designation for disease(s) considered as rare in Europe, whether they were further developed to become drugs with marketing authorisation (MA) or not.

The Orphanet database also includes drugs without an orphan designation as long as they have been granted a marketing authorisation with a specific indication for a rare disease.

The data is extracted from official sources.

Orphanet acknowledges the contribution of the European Medicines Agency (EMA) to the creation of the database on orphan medicinal products in Europe. This project was initiated by the Committee for Orphan Medicinal Products (COMP) at the EMA. Several members of the COMP have individually contributed to the development of the database, which was welcomed by the COMP as a significant tool for all stakeholders.
The database has been developed entirely and autonomously by Orphanet, using public and private funding and publicly available data; as such, neither the EMA nor the COMP are responsible for the content and management of the database.


The Orphanet database of orphan drugs was developed with co-funding from the following institutions and companies:

Leem novartis pfizer Merck Serono PharmaMar Genzyme Biomarin