“Orphan Drug” designation

“Orphan Drug”: a designation to ease research

Several products in development for the treatment of Stargardt disease have received the “Orphan Drug” designation from the European Medicines Agency (EMA) or from the Food and Drug Administration (FDA, American Healthy Agency).

These are products being studied (treatment trials) which have potential efficacy against the rare disease concerned.

Warning: It is not a medical drug in the usual sense, but it is a designation for products being studied for the treatment of a rare disease. The “orphan drug” designation is not a marketing authorisation.

The designation is given by the EMA and the FDA to support and protect investors who involve themselves in the development of medical drugs which will have a limited market of patients by definition: due to the rarity of the disease, there is less investment for treatments.

Thanks to this designation, companies involved will be able to have:

  • Advantages during development: tax credits, privileged access to funding, shorter clinical trials and more easily granted regulatory agreements.
  • Commercial leverage: fast market access, longer protection than traditional medical drugs after their market release (10 years in Europe, 7 years in the USA).

NB : Condition of rarity: a rare disease  affects:

  • Less than 5 out of 10 000 people in Europe
  • Less than 200 000 people in the USA


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