Gene therapy company receives Rare Paediatric Disease Designation for the treatment of Achromatopsia

Posted on: Wednesday 7 February 2018

MeiraGTx, a London and New York-based gene therapy company, has announced that the Offices of Orphan Products Development and Pediatric Therapeutics of the U.S. Food and Drug Administration (FDA) have granted rare paediatric disease designation to the company’s gene therapy product candidate AAV2/8-hCARp.hCNGB3 (A002) for the treatment of patients with achromatopsia due to mutations in the CNGB3 gene.

A002 previously received orphan drug designation from the FDA and the European Medicines Agency (EMA) for the treatment of ACHM in 2016.

A002 is an adeno-associated virus investigational gene therapy designed to deliver a codon-optimised hCNGB3 cDNA to the back of the eye for expression in cone photoreceptors. 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 dosed eight patients in an open label, multi-centre Phase I/II dose escalation trial of A002 in individuals diagnosed with achromatopsia due to biallelic mutations in CNGB3. The trial is currently treating patients in the highest of three dose cohorts. The primary endpoint of the study is to determine the safety of the treatment. Secondary endpoints include improvement in visual function including assessment of photophobia/photoaversion, retinal function, retinal structure and quality of life measures.

“With the receipt of our second rare paediatric disease designation in just two months, we have significant momentum for our ocular gene therapy pipeline as we begin 2018,” said Zandy Forbes, Ph.D., President and CEO of MeiraGTx. “Achromatopsia is a severe and debilitating genetic disorder of the retina for which there are no effective treatment options. We are excited to be making such good progress in dosing patients in our ongoing Phase I/II CNGB3 clinical trial.”