Azetukalner NDA Submitted, EMA Gives Approval Recommendation to Ocrelizumab, Phase 3 FUSION Trial Meets End Points

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Welcome to the Neurology News Network, my name is Louie Pasculli and here’s a look at some of the top stories in Neurology.

Beginning with FDA news, the administration Xenon Pharmaceuticals has submitted a new drug application (NDA) to the FDA for azetukalner (XEN1101) as adjunctive treatment for focal seizures in adults with epilepsy, based on data from the phase 3 X-TOLE2 trial and the earlier phase 2b X-TOLE study. If approved, azetukalner would be the first KV7 potassium channel opener available for epilepsy, with a mechanism distinct from that of currently marketed antiseizure medications.¹

The NDA was supported by more than 1500 patient-years of safety data across the azetukalner epilepsy program, with Xenon describing the safety profile as consistent and generally well tolerated across studies. The company is separately developing azetukalner in psychiatry, with topline data from the ongoing phase 3 X-NOVA2 study in major depressive disorder expected in the first quarter of 2027.

“Azetukalner has the potential to deliver much needed innovation to treat people living with focal seizures, with strong efficacy, a differentiated mechanism of action that may enable rational polytherapy, once-daily dosing with no dose adjustments for other antiseizure medications, and a consistent and generally well-tolerated safety profile,” Ian Mortimer, president and CEO of Xenon, said in a statement.¹

In a new company update, Roche announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended approval of Ocrevus (ocrelizumab) intravenous infusion for pediatric patients aged 10 years and older with relapsing forms of multiple sclerosis (RMS). The recommendation follows findings from the phase 3 OPERETTA 2 study, which evaluated ocrelizumab against fingolimod in pediatric patients with relapsing-remitting MS.²

The European Commission (EC) is expected to issue a final decision following the CHMP’s positive opinion. Earlier, in May, the FDA approved ocrelizumab for pediatric patients with relapsing MS in the United States.

“Children and teens living with multiple sclerosis experience more frequent and severe relapses than adults, yet their treatment options have lagged behind,” Levi Garraway, MD, PhD, chief medical officer and head of global product development at Roche, said in a statement.² “The positive CHMP opinion brings us closer to bridging a longstanding gap in Europe, offering young people with MS a high-efficacy therapy backed by a decade of adult experience.”

In other news, Otsuka Pharmaceutical and Ionis Pharmaceuticals announced positive topline results from the phase 3 FUSION trial (NCT04768972) evaluating ulefnersen, an investigational antisense oligonucleotide (ASO), in patients with a rare genetic subtype of amyotrophic lateral sclerosis (ALS) driven by mutations in the fused in sarcoma (FUS) gene, known as FUS-ALS.³

According to the companies, the trial met its primary end point, showing a statistically significant improvement on a composite measure of functional decline and survival, marking the first placebo-controlled clinical evidence for an agent designed to target the underlying genetic cause of the disease. Otsuka and Ionis noted plans to share full results with the FDA and other regulators and to present detailed findings at a future medical congress and in a peer-reviewed publication.

“We are profoundly grateful to the patients, carers, families, investigators and study teams whose commitment made this study possible,” Kunal Gandhi, MD, MPH, Vice President, Head of Global Clinical Development, at Otsuka, told NeurologyLive^®. “These findings represent a major advancement for people living with FUS-ALS, a rare and often rapidly progressive genetic form of ALS. There are currently no approved therapies specifically targeting the underlying genetic cause of FUS-ALS, underscoring the significant unmet need for effective treatment options. As the first FUS-ALS clinical trial to meet its primary endpoint, FUSION provides compelling evidence that a targeted genetic approach may help alter the course of disease.”

To read the full pieces and to get more direct access to expert insight, head to NeurologyLive.com. Be sure to tune in next week to remain informed on the latest in neurology. I’m Louie Pasculli, thanks for watching Neurology News Network.

REFERENCES
1. Xenon announces azetukalner NDA submission to FDA for focal seizures. News release. Xenon Pharmaceuticals. September 17, 2026. Accessed September 21, 2026. https://investor.xenon-pharma.com/news-releases/news-release-details/xenon-announces-azetukalner-nda-submission-fda-focal-seizures
2. CHMP recommends EU approval of Roche’s Ocrevus for children and adolescents with relapsing multiple sclerosis. Roche. News Release. September 18, 2026. Accessed September 21, 2026. https://www.globenewswire.com/news-release/2026/09/18/3364612/0/en/chmp-recommends-eu-approval-of-roche-s-ocrevus-for-children-and-adolescents-with-relapsing-multiple-sclerosis.html_gl=1*1u33qxf*_up*MQ..*_ga*NDAxNTQ4NC4xNzg5OTIyODI0*_ga_B6167QB2TF*czE3ODk5MjI4MjMkbzEkZzAkdDE3ODk5MjI4MjMkajYwJGwwJGgxNTg1MzIyNDMz*_ga_ERWPGTJ5X8*czE3ODk5MjI4MjMkbzEkZzAkdDE3ODk5MjI4MjMkajYwJGwwJGg
3. Otsuka Announces Transformative Phase 3 FUSION Results for Ulefnersen, Bringing the FUS-ALS Community Closer to a Potential Targeted Treatment. News release. Otsuka. September 22, 2026. Accessed September 22, 2026. https://www.otsuka-us.com/news/otsuka-announces-transformative-phase-3-fusion-results-ulefnersen-bringing-fus-als-community