20 August 2018 - MeiraGTx today announced that the U.S. FDA has granted fast track designation for its AAV-CNGB3 gene therapy product candidate for the treatment of achromatopsia caused by mutations in the CNGB3 gene.

AAV-CNGB3 has been granted orphan drug designation by the FDA and orphan medicinal product designation by the EMA, as well as rare paediatric 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 paediatric patients, with 14 patients treated to date. Eleven adult patients have been treated in three dose escalation cohorts and three paediatric patients have now been treated in the extension phase of the study. The company anticipates completing dosing of up to eight paediatric patients in the second half of 2018.

Read MeiraGTx press release