FDA grants Regenerative Medicine Advanced Therapy designation to epilepsy treatment

The FDA has granted Regenerative Medicine Advanced Therapy designation to a novel regenerative cell therapy to treat drug-resistant mesial temporal lobe epilepsy, according to the manufacturer.

“We are excited to receive this RMAT designation from the FDA and look forward to working with the agency to most efficiently evaluate NRTX-1001 for people with drug-resistant focal epilepsy,” Cory R. Nicholas, PhD, co-founder and CEO of Neurona Therapeutics, told Healio in an email. “We are encouraged by the preliminary data from the ongoing open label phase 1/2 clinical trial in unilateral temporal lobe epilepsy, and we are now actively enrolling a second open label study in bilateral temporal lobe epilepsy.”

In a press release, Neurona said NRTX-1001 is being evaluated in a pair of open-label multicenter phase 1/2 clinical trials to examine safety and efficacy of a single administration of the therapy for drug-resistant unilateral mesial temporal lobe epilepsy (unilateral MTLE) and for drug-resistant bilateral mesial temporal lobe epilepsy (bilateral MTLE). The first stage of the trial will be an open-label dose-escalation study in 10 participants, recently expanded to at least 16 enrollees, with eight patients to be treated at a starting dose and eight others at a higher dose.

NRTX-1001 is a regenerative cell therapy derived from human pluripotent stem cells. According to the release, these fully differentiated neural cells, called interneurons, secrete the inhibitory neurotransmitter gamma-aminobutyric acid (GABA) and are intended to provide long-term GABAergic inhibition to repair hyper-excitable neural networks.

With continued positive data from the ongoing studies, NRTX-1001 is likely to be investigated to treat neocortical focal epilepsy and other indications such as Alzheimer’s disease, Nicholas said.