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List of Orphan Designations

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The Orphan Designation is a legal procedure that allows for the designation of a medicinal substance with therapeutic potential for a rare disease, before its first administration in humans or during its clinical development.
The exact therapeutic indication is then defined at the time of marketing authorisation.
This procedure has been established in Europe by the Regulation (EC) No 141/2000
Similar procedures exist in the US, Australia, Japan and Singapore.


Choose a letter from the alphabetical list and click on it in order to obtain substances with at least an orphan designation. The concerned geographical area(s) is(are) indicated.

Then click on the substance name to access to the “Substance” sheet or click on the geographical area in order to obtain detailed information on the designation(s) in that particular area.

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 data is extracted from official sources.

You can also access European or American designation data, respectively, through these two links : (USA)

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

Orphan Designations by geographical area