3CB8968Orphan drugs are intended to treat diseases so rare that sponsors are reluctant to develop them under usual marketing conditions. They may be defined as drugs that are not developed by the pharmaceutical industry for economic reasons but which respond to public health need. The indications of a drug may also be considered as ' orphan ' since a substance may be used in the treatment of a frequent disease but may not have been developed for another, more rare indication.

An orphan designation allows a pharmaceutical company to benefit from incentives from the European Union to develop a medicine for a rare disease, such as reduced fees and protection from competition once the medicine is placed on the market. Applications for orphan designation are examined by the COMP (Committee for Orphan Medicinal Products), which adopts an opinion that is forwarded to the European Commission. The European Commission then decides whether to grant an orphan designation for the medicine in question. 

Rare eye disorders have a significant health impact as more than 60 % of childhood cases of blindness are due to congenital cataracts and glaucoma, retinal degenerations, optic atrophy, and eye malformations. Some of the rare retinal diseases such as retinitis pigmentosa, Usher syndrome (a deaf–blindness condition), Stargardt's disease, Leber's congenital amaurosis, etc., are within the main scientific interests of the Institut de la Vision.

Sources : Orphanet, European Medicines Agency


Inventory of orphan drug designations for treatment of eye diseases

From the Community Register of orphan medicinal products for human use