MeiraGTx says AAV-CNGB3 granted fast track designation for ACHM treatment
MeiraGTx Holdings announced that the U.S. FDA has granted Fast Track designation for its AAV-CNGB3 gene therapy product candidate for the treatment of achromatopsia, or ACHM, caused by mutations in the CNGB3 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, Ph.D., 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 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. In April 2018, the company was also granted Fast Track designation for AAV-RPGR for the treatment of another debilitating ocular disorder, X-linked retinitis pigmentosa due to defects in the RPGR gene.