Rare Disease Report

Achromatopsia Treatment, AAV-CNGA3, Granted Rare Pediatric Disease Designation

AUGUST 27, 2018
Krista Rossi
The US Food and Drug Administration (FDA) has granted a rare pediatric disease designation to MeiraGTx Holdings Plc’s AAV-CNGA3 for the treatment of patients with achromatopsia (ACHM) due to mutations in the CNGA3 gene.

AAV-CNGA3 is a gene therapy engineered to restore cone function in eye cells; it is administered through subretinal injection, which allows treatment delivery to the cone receptors at the back of the eye.

“Without any currently approved therapies, we are very pleased by the FDA’s decision and the recognition from the Agency that those suffering from ACHM are in need of urgent treatment options,” said Alexandria Forbes, PhD, president and chief executive officer of MeiraGTx, in a statement regarding the recent orphan drug designation granted to AAV-CNGA3 by the FDA earlier this August. “This designation is the second important regulatory milestone we’ve received for AAV-CNGA3 in just 2 months and we look forward to continuing the momentum in this program for those in need of relief from this debilitating disease.”

A rare inherited retinal disease, ACHM severely limits an individual’s vision due to its inhibition of the function of cone photoreceptors. Patients with ACHM typically have extreme sensitivity to light, experience involuntary eye movements, and are legally blind from birth.

In addition to the recent FDA orphan drug designation, the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products gave a positive opinion on AAV-CNGA3, recommending an orphan drug designation for AAV-CNGA3 for the treatment of ACHM in June 2018.  
 

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