MeiraGTx Announces AAV-CNGA3 Granted Fast Track Designation by FDA for Treatment of Achromatopsia

MeiraGTx Holdings announced that the FDA has granted Fast Track designation to its AAV-CNGA3 gene therapy product candidate for the treatment of achromatopsia (ACHM) caused by 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 and have extremely debilitating sensitivity to light. 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.

MeiraGTx and Janssen Pharmaceuticals (Johnson & Johnson) are jointly developing AAV-CNGA3 as part of a broader collaboration to develop and commercialize gene therapies for the treatment of inherited retinal diseases. MeiraGTx is currently conducting an open-label, dose-escalation phase 1/2 clinical trial of AAV-CNGA3 in patients with ACHM due to mutations in the CNGA3 gene. (NCT03758404)

“We are very pleased to have received Fast Track designation for AAV-CNGA3 and that the FDA has recognized a significant need exists to quickly advance new therapies for those with ACHM,” Alexandria Forbes, PhD, president and chief executive officer of MeiraGTx, said in a company news release. “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.”

The FDA’s Fast Track process is designed to expedite the development and review of drugs used to treat serious conditions and fill an unmet medical need. Fast Track designation enables the company to have early and frequent communication with the FDA throughout the drug development and review process, with the potential for faster drug approval and patient access.

AAV-CNGA3 has already been granted Orphan Drug designation by the FDA and European Medicines Agency (EMA). In addition, AAV-CNGA3 for the treatment of ACHM caused by mutations in the CNGA3 gene has been designated as a drug for a rare pediatric disease by the FDA.

This is the third Fast Track designation MeiraGTx has received for its inherited retinal disease product candidates, having previously received Fast Track designation for AAV-RPGR and AAV-CNGB3, as well as having received PRIME designation for AAV-RPGR and AAV-CNGB3 from the EMA. AAV-CNGB3 for the treatment of ACHM caused by mutations in the CNGB3 gene has also been designated as a drug for a rare pediatric disease by the FDA.

MeiraGTx also recently received orphan drug designation from the FDA as well as orphan medicinal product designation from the EMA for its investigational gene therapy product AAV-RDH12 for the treatment of retinol dehydrogenase 12 (RDH12) mutation-associated retinal dystrophy.

Disease-causing sequence variants in RDH12 cause severe retinal dystrophy most often resulting in the clinical diagnosis of Leber congenital amaurosis (LCA) and early onset severe retinal dystrophy (EOSRD); although RDH12 variants have also been associated with a clinical diagnosis of retinitis pigmentosa (RP).  Sequence variants in RDH12 account for 3.4%–10.5% of LCA/EOSRD. Individuals with RDH12 deficiency exhibit widespread retinal degeneration impacting both rods and cones, with early macular involvement. Most people with RDH12–LCA/EOSRD experience marked central visual loss by their late teens to twenties.  AAV-RDH12 is an AAV based gene therapy designed to deliver a functional copy of the RDH12 gene to the retina of patients with genetically defined RDH12 deficiency.

About Achromatopsia

Achromatopsia (ACHM) is an inherited retinal disorder that specifically prevents cone photoreceptors from functioning. ACHM is characterized by severely reduced visual acuity of 20/200 or worse, reduced or complete loss of color vision, disabling light sensitivity (photoaversion) and involuntary back and forth eye movements (nystagmus). ACHM occurs in approximately one in 30,000 people in the United States, with 92 percent of cases caused by mutations in CNGB3 and CNGA3 genes. Currently, there are no effective treatments for this disease.