October 22, 2015 8:37am


 

The European Medicines Agency (EMA) has granted an orphan drug designation to its investigational gene therapy product for the treatment of achromatopsia caused by mutations in the CNGA3 gene.

 

AGTC is developing products for achromatopsia based on mutations in the CNGB3 and CNGA3 genes, which together account for 75% of the total achromatopsia patient population.

AGTC has demonstrated proof-of-concept in a naturally occurring dog model of the CNGB3 form of the disease, and previously received orphan drug designation from the U.S. Food and Drug Administration and the EMA for its investigational gene therapy product for the treatment of achromatopsia caused by mutations in the CNGB3 gene.

 

The Bottom Line: More recent studies conducted by AGTC's collaborators showed that in ---sheep affected by achromatopsia caused by mutations in the CNGA3 gene, delivery of an AAV vector carrying a normal copy of CNGA3 restored vision as measured by the ability to navigate an obstacle maze. Receiving EU orphan drug designation is an important milestone that delivers significant commercial benefits to any company.

 

AGTC closed at $12.65 which was DOWN -$0.02 on small volume - BUY

 

Achromatopsia is an inherited retinal disease, which is present from birth and is characterized by the lack of cone photoreceptor function. The condition results in markedly reduced visual acuity, extreme light sensitivity causing day blindness, and complete loss of color discrimination. Best-corrected visual acuity in persons affected by achromatopsia, even under subdued light conditions, is usually about 20/200, a level at which people are considered legally blind. The incidence rate for achromatopsia is approximately one in 30,000 people, and it is estimated that there are approximately 10,000 people in the United States and 17,000 people in Europe with achromatopsia.