Gene Signal

Orphan Drugs

The European Medicines Agency’s (EMA) 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 Agency’s (EMA) has granted Gene Signal four Orphan Drug designations in Ophthalmology

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

On 11 June 2014, Orphan designation (EU/3/14/1275)  was granted by the European Commission to Gene Signal for aganirsen (GS-101) for the treatment of Central Retinal Vein Occlusion, the most common cause of Neovascular Glaucoma. 

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 

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