Gene therapies developer MeiraGTx has obtained rare paediatric disease designation from the US Food and Drug Administration (FDA) for its new product candidate AAV2/5-OPTIRPE65 (A001) to treat Leber’s Congenital Amaurosis caused by RPE65 gene mutations (LCA2).

LCA is a series of autosomal recessive, early-onset retinal dystrophies that could result in sight impairment during childhood.

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A001 is an investigational adeno-associated virus (AAV2/5) gene therapy being developed to provide a codon-optimised RPE65 cDNA controlled under a synthetic RPE-specific promoter.

The therapy will be delivered to the back of the eye through a subretinal injection. FDA granted orphan drug designation to the product candidate last year for treating LCA2.

“We are pleased with the rapid progress in our LCA2 clinical study and we are excited to be including paediatric patients with this severe genetic disorder in both Europe and the US.”

MeiraGTx president and CEO Zandy Forbes said: “The FDA’s decision to award rare paediatric disease designation to A001, along with the previously received orphan drug designation, underscores the urgency of developing effective therapies for rare paediatric diseases like LCA2.

“We are pleased with the rapid progress in our LCA2 clinical study and we are excited to be including paediatric patients with this severe genetic disorder in both Europe and the US.”

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A001 is being currently studied in an open-label, multi-centre, dose-escalation Phase I/II clinical trial in approximately 27 patients aged three years and above.

The trial’s primary endpoint is safety, while the secondary endpoints include improved visual function, retinal function, retinal structure and quality of life measures.

MeiraGTx received FDA approval for A001’s investigational new drug (IND) application in October this year.