FDA Grants Fast Track Designation to MeiraGTx for Experimental Retinal Disease Gene Therapy

Rare Daily Staff

The U.S. Food and Drug Administration granted Fast Track designation to MeiraGTx for its AAV-CNGB3 experimental gene therapy candidate for the treatment of the retinal disease achromatopsia caused by mutations in the CNGB3 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-CNGB3 is an experimental gene therapy treatment designed to restore cone function, delivered to the cone receptors at the back of the eye via subretinal injection.

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. Today’s announcement marks the second Fast Track designation granted to MeiraGTx in 2018. In April, the FDA granted Fast Track designation for the company’s AAV-RPGR for the treatment of another eye disorder, X-linked retinitis pigmentosa due to defects in the RPGR gene.

“We are thrilled by this designation and the continued recognition by the FDA of AAV-CNGB3’s potential to become a much-needed treatment option for ACHM patients suffering from this debilitating disease,” said Alexandria Forbes, president and CEO of MeiraGTx. “Fast Track designation is not just a milestone for the company, but also for the ACHM community. With the ability to communicate closely and often with the FDA, we are hopeful that we can expedite our commitment to bring a safe and effective therapy to patients in need.”

AAV-CNGB3 has also been granted orphan drug designation by the FDA and orphan medicinal product designation by the European Medicines Agency, as well as rare pediatric disease designation by the FDA and PRIME designation by the EMA, for the treatment of ACHM caused by mutations in the CNGB3 gene.

MeiraGTx is currently conducting a phase 1/2 clinical trial of AAV-CNGB3 in both adult and pediatric patients, with 14 patients treated to date. Eleven adult patients have been treated in three dose escalation cohorts and three pediatric patients have now been treated in the extension phase of the study. The company anticipates completing dosing of up to eight pediatric patients in the second half of 2018.

August 20, 2018
Photo: Alexandria Forbes, president and CEO of MeiraGTx