MeiraGTx reports Fast Track designation by FDA for gene therapy candidate to treat ACHM

MeiraGTx Holdings Plc announced today that the Food and Drug Administration (FDA) has granted Fast Track designation to its AAV-CNGA3 gene therapy product candidate for the treatment of achromatopsia (ACHM).

ACHM, an inherited retinal disease caused by mutations in the CNGA3 gene, severely limits a person’s vision by preventing cone photoreceptors in the eye from functioning.

AAV-CNGA3 is designed to restore cone function by delivering to the cone receptors located in the back of the eye with subretinal injection, according to the company.

The treatment uses a synthetic promoter associated with strong gene expression to make up for the large protein quantity needed to restore cone function in ACHM patients with the CNGA3 gene mutation.

The investigational gene therapy treatment was jointly developed by MeiraGTx and Janssen Pharmaceuticals (of Johnson and Johnson) as part of a broader partnership to develop and commercialize gene therapies for inherited retinal disease treatment, the release stated.

“ACHM is a serious and debilitating disease and we look forward to communicating closely with the FDA as we continue the clinical advancement of AAV-CNGA3,” said Alexandria Forbes, PhD, president and CEO of MeiraGTx, in the release.

MeiraGTx is also currently conducting a phase ½ clinical trial for AAV-CNGA3 in pediatric patients with ACHM. The gene therapy treatment was previously granted Orphan Drug designation by the FDA and European Medicines Agency (EMA) as well as Rare Pediatric Disease (RPD) designation by the FDA.¹

References
1. MeiraGTx. Research and Development. https://meiragtx.com/research-development/pipeline/