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Orphan diseases listing

There is currently no website that provides a list of orphan drugs available for purchase on the market, whether in Europe or globally.

Orphan Drugs authorised to be marketed

Europe

The Enterprise DG (Directorate General) of the European Commission offers a list of all designated orphan drugs for the European market.

The others are still under development or awaiting authorisation.

Before a drug can be marketed and be available for the patients in a specific country in Europe, however, it may also require additional administrative clearance by the country's authorities (setting of price and public health insurance reimbursement tariffs, for example).

The Enterprise DG list does not provide in-depth details on the drug. However, it provides a trade name for the drug, which you can then use to find in-depth information about the product on the website of the EMA (European Medicines Agency) The EMA listing covers all drugs, not just orphan drugs. Information is available in all EU languages.

Orphan Drugs waiting to come to market

(These drugs are labelled or "designated" as orphan drugs, but are not yet authorised for sale)

Europe

European Union registry of designated orphan drugs

For each drug, there is a "Summary of Opinion" that gives details about the drug./>

A Summary describes:

  • Drug name
  • Chemical description of the drug (for some drugs)
  • Disease intended to treat
  • Current methods of treatment for the disease
  • Estimated number of patients affected by the disease in Europe
  • Expected action of the drug
  • Developmental stage of the drug
  • Contact information for the pharmaceutical sponsor of the drug
  • Contact information for a European patient organisation concerned by the disease (if available)
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