China’s Center for Drug Evaluation (CDE) has awarded breakthrough therapy designation (BTD) to Wuhan Neurophth Biological Technology Co., Ltd’s NR082 for the treatment of Leber hereditary optic neuropathy (LHON) caused by the G11778A mutation. The drug, an innovative intraocular injection gene therapy, uses a recombinant adeno-associated virus serotype 2 vector (rAAV2).
Mechanism and Development
NR082 delivers the correct gene to damaged optic ganglion cells via a single intravitreal injection, repairing the mitochondrial respiratory chain and restoring optic nerve function. The therapy has demonstrated safety and efficacy in three investigator-initiated trials (ITTs), with some patients showing benefits for up to 90 months.
Regulatory Milestones
In March 2021, NR082 became the first in vivo ophthalmology gene therapy to receive clinical trial approval in China. A clinical study commenced two months later, marking a significant step in the development of gene therapies for LHON.-Fineline Info & Tech
