Gene Signal

Orphan Drugs

In the European Regulation, Orphan Drugs are defined 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 (EMA) has granted Gene Signal four Orphan Drug designations to aganirsen in Ophthalmology:

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 aganirsen (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 aganirsen (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 aganirsen (GS-101) for the treatment of Retinopathy of Prematurity.

In the United States, Orphan Drugs are defined as follows: Drug intended for a rare disease or condition. The term "rare disease or condition'' means any disease or condition which: affects less than 200,000 persons in the United States, or affects more than 200,000 in the United States and for which there is no reasonable expectation that the cost of developing and making available in the United States a drug for such disease or condition will recovered from sales in the United States of such drug.

On November 7, 2016, the US Food and Drug Administration granted aganirsen an Orphan Drug Designation for Prevention of Corneal Graft Rejection.