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List of Substances

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This list contains all substances which are components of Drugs with an indication for a rare disease, whether they have been granted an orphan designation or not (for more information on the orphan designation procedure, see List of Orphan Designations).

Choose a letter in the alphabetical list and click on it in order to obtain corresponding substances and associated trade name(s) when applicable.

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 (MA) 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

426 Result(s)