Orphanet provides an inventory of drugs at all stages of development for one particular rare disease or a group of rare diseases. This includes all the substances which have been granted an orphan designation for disease(s) considered as rare in Europe or the USA, whether they were further developed to become approved 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.
Some drugs are included in the database because they are tested in a clinical trial performed on a rare disease, but they do not have a regulatory status.
Search by disease name: Enter your requested disease name to access the list of medicinal products for this disease. Search by substance/trade name: Enter your requested drug by its commercial name (Trade name) or substance name including chemical names, code names of the substance or common names (e.g. INN : International Non-proprietary Name).
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 on Orphan Medicinal Products (EC) No 141/2000 and in the US by the Orphan Drug Act.
Information in Orphanet is updated on a regular basis. It may happen that there is a delay between the granting of a new designations/approvals and their publication in Orphanet. Professionals are always encouraged to consult the most recent publications before making any decisions based on the information provided.
Orphanet cannot be held responsible for harmful, truncated or erroneous use of any information found in the Orphanet database.
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.