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List of Orphan Drugs with MA

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Orphan Drugs are medical products with an orphan designation (for more information on the orphan designation procedure, see List of Orphan Designations), and a marketing authorisation associated, in Europe, with a positive assessment of significant benefice.


Choose a letter in the alphabetical list and click on it in order to obtain the trade names of drugs with at least a marketing authorisation with an orphan designation. The concerned geographical area(s) is(are) indicated. Then click on the trade name to access to the “Drug” sheet or click on the geographical area in order to obtain detailed information on the market authorisation 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 Drugs by geographical area