MeiraGTx Holdings Plc has announced that the U.S. Food and Drug Administration (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, Inc. (Janssen), one of the Janssen Pharmaceutical Companies of 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,” said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx. “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.
Full press release: investors.meiragtx.com/news-releases/news-release-details/meiragtx-announces-aav-cnga3-granted-fast-track-designation-us