The European Medicines Evaluation Agency’s (EMEA) has defined Orphan Drugs as follows

A medicinal product is designated as an orphan medicinal product if:

• It is intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition affecting no more than 5 in 10,000 persons in the European Union at the time of submission of the designation application (prevalence criterion), or
• It is intended for the diagnosis, prevention or treatment of a life-threatening, seriously debilitating or serious and chronic condition and without incentives it is unlikely that expected sales of the medicinal product would cover the investment in its development, and
• No satisfactory method of diagnosis, prevention or treatment of the condition concerned is authorised, or, if such method exists, the medicinal product will be of significant benefit to those affected by the condition.

The European Medicines Evaluation Agency’s (EMEA) has granted Gene Signal three Orphan Drug designations in Ophthalmology

The following Orphan Drug designations were granted to Gene Signal for GS 101 - antisense oligonucleotide:

On October 2nd 2003, Orphan designation (EU/3/03/161) was granted by the European Commission to Gene Signal for GS-101 for the treatment of Neovascular Glaucoma

On October 2nd 2003, Orphan designation (EU/3/03/160) was granted by the European Commission to Gene Signal for GS-101 for the treatment of Retinopathy of Prematurity

On 17 April 2007, Orphan designation (EU/3/07/445) was granted by the European Commission to Gene Signal for GS-101 for the prevention of Corneal Graft Rejection 

Protocol for I- GRAFT  - Gene Signal’s  multicentre double-blind randomized study investigating the efficacy and tolerability of GS-101 eye drops for the inhibition of corneal neovascularisation  - now on Orphanet

The protocol for Gene Signal’s Phase III  I- GRAFT study is now published on Orphanet, the portal for rare diseases and Orphan Drugs.  I-GRAFT is a multicentre double-blind randomized study to investigate the efficacy and tolerability of GS-101 eye drops, an antisense oligonucleotide, versus placebo on inhibition of corneal neovascularisation, a major risk factor of corneal graft rejection.