Gene therapy company MeiraGTx Holdings announced that the FDA has granted rare pediatric disease designation to the company’s gene therapy product candidate AAV-CNGA3 for the treatment of patients with achromatopsia (ACHM) due to mutations in the CNGA3 gene.
ACHM is an inherited retinal disease that severely limits a person’s sight by preventing cone photoreceptors in the eye from functioning. Individuals with ACHM are often legally blind from birth, have extreme sensitivity to light, and experience involuntary eye movements. 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.
“We are very pleased that the FDA and EMA continue to recognize the important research MeiraGTx is doing for people living with rare inherited disorders such as ACHM,” Zandy Forbes, PhD, chief executive officer of MeiraGTx. “Our focus remains on patient benefit and developing products that offer a cure to people living with serious diseases. Working closely with expert clinicians and the FDA, we look forward to bringing impactful therapies to those in need.”
In 2018, AAV-CNGA3 was granted orphan drug designation (ODD) by the FDA and received a positive opinion from the European Medicines Agency’s (EMA) Committee for Orphan Medicinal Products recommending orphan medicinal product designation. Both the ODD and EMA positive opinion for AAV-CNGA3 are indicated for the treatment of ACHM caused by mutations in the CNGA3 gene.
A rare pediatric disease designation may be granted by the FDA to drugs and biologics intended to treat certain orphan diseases affecting fewer than 200,000 patients in the U.S., the serious or life-threatening manifestations of which primarily affect individuals aged 18 years or younger. Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor that receives approval for a biologics license application for a rare pediatric disease may be eligible to receive a voucher for a priority review of a subsequent marketing application for a different product.