NeuroPace Files FDA Application for RNS System Expansion to Treat Drug-Resistant Generalized Epilepsy
Key Insights
NeuroPace has filed a PMA supplement with the FDA seeking to expand its RNS System (search) indication to include patients with drug-resistant idiopathic generalized epilepsy (search) (IGE) with generalized tonic-clonic seizures (search).
The application is supported by positive 18-month NAUTILUS trial data showing a 77% median reduction in generalized tonic-clonic seizures (search) with favorable safety profile.
The RNS System (search) has received Breakthrough Device Designation for this IGE indication, addressing a significant unmet medical need as no approved neuromodulation options currently exist for this patient population.
NeuroPace has filed a Premarket Approval Supplement (PMA-S) application with the U.S. Food and Drug Administration seeking to expand the labeled indication for its RNS System (search) to include patients with antiseizure-medication resistant idiopathic generalized epilepsy (search) (IGE) with generalized tonic-clonic seizures (search). The submission represents a significant milestone in addressing an unmet medical need for patients who currently have no approved neuromodulation treatment options.
Strong Clinical Evidence Supports Regulatory Filing
The PMA-S is supported by positive preliminary results from the NAUTILUS trial, the first randomized controlled trial of neuromodulation in drug-resistant idiopathic generalized epilepsy (search). At 18 months of therapy, patients treated with the RNS System (search) experienced a 77% median reduction in generalized tonic-clonic (GTC) seizures compared with baseline (p<0.001). The reductions in GTCs and days without any generalized seizure were rapid and sustained over time.
"Seeing a 77% median reduction in GTCs at 18 months of therapy in this refractory population is remarkable," said Martha Morrell, MD, Chief Medical Officer of NeuroPace. "Notably, the 18-month median seizure reduction in the studied IGE population meaningfully exceeds what was observed in our original pivotal focal trial, despite the differences in patient populations and seizure types."
The clinical benefits were strongly endorsed by both physicians and patients, with Clinical Global Impression of Change exceeding 80% in both groups at 18 months, underscoring the clinical meaningfulness of the observed benefit. Safety results were consistent with the well-established RNS System (search) profile and demonstrated highly significant outcomes (p<0.0001).
Breakthrough Designation Facilitates FDA Review
The RNS System (search) has been granted Breakthrough Device Designation for this IGE indication, reflecting the significant unmet clinical need and the potential for the therapy to provide more effective treatment than existing options. Under current FDA guidelines, PMA supplements are typically reviewed within approximately 180 days, although actual review times may vary.
"Filing the PMA supplement for idiopathic generalized epilepsy (search) is an important milestone in our strategy to make responsive neuromodulation available to more patients who need it," said Joel Becker, President and Chief Executive Officer of NeuroPace. "Unlike focal epilepsy, patients with drug-resistant IGE have no approved alternative treatment options, and the NAUTILUS data show that RNS therapy can deliver robust and clinically meaningful seizure reductions with an excellent safety profile."
Addressing Critical Treatment Gap
Approximately 30% of people with idiopathic generalized epilepsy (search) continue to have disabling generalized tonic-clonic seizures (search) despite multiple antiseizure medications (search), and there are no approved neuromodulation or surgical options for these patients. If approved, the RNS System (search) would become the first neuromodulation therapy indicated for idiopathic generalized epilepsy.
"These data strengthen our conviction that the RNS System (search) has the potential to become a foundational adjunctive therapy for drug-resistant idiopathic generalized epilepsy (search)," Becker noted. "The preliminary 18-month NAUTILUS results further demonstrate that the RNS System has provided evidence of a compelling risk benefit profile that could result in the first neuromodulation therapy indicated for idiopathic generalized epilepsy."
The RNS System (search) is currently the first and only commercially available, brain-responsive platform that delivers personalized, real-time treatment at the seizure source. The technology has the potential to drive a better standard of care for patients living with drug-resistant epilepsy (search) and offer improved outcomes to the large population of patients suffering from various brain disorders.