MeiraGTx Receives EMA PRIME Designation for Achromatopsia Gene Therapy Candidate

Posted on: Monday 5 March 2018

MeiraGTx, a London and New York based gene therapy company, has announced the European Medicines Agency (EMA) has granted Priority Medicines (PRIME) designation to its gene therapy product candidate A002 for the treatment of patients with achromatopsia due to mutations in the CNGB3 gene. PRIME is an initiative launched by EMA in 2016 to enhance support for the development of medicines that target an unmet medical need, and may offer a major therapeutic advantage over existing treatments, or benefit patients without treatment options. PRIME aims to strengthen clinical trial designs to facilitate the generation of high quality data for the evaluation of an application for marketing authorization. To be accepted for PRIME, a medicine has to show its potential to benefit patients with unmet medical needs based on preclinical and / or early clinical data. The PRIME application was based on non-clinical in vivo data and early clinical safety data from MeiraGTx’s ongoing Phase I / II dose escalation study.

“With receipt of this important designation, we are excited to be begin working closely with the EMA to accelerate A002 as a potential treatment option for patients living with this challenging and painful condition,” said Zandy Forbes, Ph.D., President and CEO of MeiraGTx. “Having just received Rare Paediatric Disease Designation from the U.S. Food and Drug Administration (FDA) earlier this month, we are very pleased with the momentum of our ACHM program and the recognition by regulatory agencies of its potential to help those in need of effective treatment options.”

A002 is an adeno-associated virus (AAV) investigational gene therapy designed to deliver a codon-optimised CNGB3 cDNA under the control of the cone arresting (CAR) promoter to photoreceptors in the back of the eye. A002 is delivered via a sub-retinal injection to cover the central region of the retina, including the fovea where the majority of cones are located. MeiraGTx has completed dosing of 8 CNGB3 patients in the Phase I/II study and is currently treating patients in the third dose escalation cohort. A002 received rare paediatric disease designation for the treatment of ACHM in January 2018 and has also received orphan drug designation from the FDA and the EMA.