MeiraGTx Receives Orphan Drug Designation from the U.S. FDA for the Treatment of Achromatopsia

Posted on: Wednesday, August 15th, 2018



MeiraGTx has announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) for its AAV-CNGA3 gene therapy product candidate for the treatment of achromatopsia (ACHM) caused by mutations in the CNGA3 gene.

The FDA’s Orphan Drug Designation programme provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases and disorders that affect fewer than 200,000 people in the US or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.

AAV-CNGA3 is an investigational gene therapy treatment designed to restore cone function, delivered to the cone receptors at the back of the eye via subretinal injection.

“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, Ph.D., president and chief executive officer of MeiraGTx. “This designation is the second important regulatory milestone we’ve received for AAV-CNGA3 in just two months and we look forward to continuing the momentum in this program for those in need of relief from this debilitating disease.”