Balt's Squid Receives Premarket Approval, Pitolisant Tablets Granted Extended Approval Indication, I-ACQUIRE Displays Promising Short-Term Outcomes

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Welcome to the Neurology News Network. My name is Louie Pasculli. Here’s a look at this week’s top stories in neurology.

We kick off out coverage with some FDA news. Balt Inc. announced that the FDA has granted premarket approval for the Squid liquid embolic agent for embolization of the middle meningeal artery (MMA) as an adjunct to usual care in patients with large symptomatic chronic subdural hematomas (CSDH). The approval was based on results from the prospective, randomized STEM trial, which evaluated MMA embolization for cSDH and demonstrated a significant reduction in treatment failures in a large patient population. The STEM trial, originally published in the New England Journal of Medicine in November of 2024, demonstrated that adjunctive embolization of the MMA with Squid significantly reduced treatment failure rates without increasing adverse events in patients managed with either surgery or medical management. David Fiorella, MD, PhD, STEM trial principal Investigator and professor at the Stony Brook University Medical Center in Pennsylvania, said this in a statement. “It was clear from the initial cases that Dr. Adam Arthur and I performed that middle men-in-geal artery embolization showed promise for these patients; however, high quality data were required to confirm the effectiveness and safety of the procedure and to ultimately generate a paradigm shift towards this minimally invasive treatment becoming the standard of care. I am very thankful that Balt’s leadership team aligned with this vision and took the bold step to support STEM, the very first prospective randomized trial assessing the effect of liquid embolics in patients with symptomatic CSDH.”

Staying with the FDA, the administration has granted an expanded approval indication to Harmony Biosciences’ pitolisant tablets to include the treatment of cataplexy in pediatric patients aged 6 years and older with narcolepsy. With this decision, pitolisant becomes the only FDA-approved nonscheduled therapy indicated for both pediatric and adult patients with narcolepsy, regardless of the presence of cataplexy. Pitolisant, a selective histamine H3 receptor inverse agonist, was first approved by the FDA in August 2019 for the treatment of excessive daytime sleepiness (EDS) in adult patients with narcolepsy. The approval was subsequently expanded in October 2020 to include the treatment of cataplexy in adult patients with narcolepsy, and most recently in June 2024 the indication was further broadened to include treatment of EDS in pediatric patients aged 6 years and older with narcolepsy. At the time of the expanded indication in 2024, the FDA handed Harmony a complete response letter for pitolisant as a treatment of cataplexy in this pediatric population. Narcolepsy patient advocate Julie Flygare, JD, president and CEO at Project Sleep, told NeurologyLive® quote “Given that narcolepsy often onsets in children and young adults, having more FDA-approved treatment options for this younger population is absolutely critical. Finding a good treatment regimen that works well for each child and teen allows them their greatest chance at getting through school and pursuing their dreams.”

We wrap up our coverage with Late-breaking data from a large-scale, phase 3 study. The investigation showed that high doses of I-ACQUIRE, a constraint-induced movement therapy or CIMT, led to clinically important gains in toddlers with perinatal arterial ischemic stroke or PAIS. At 6 months after the conclusion of treatment, exploratory findings showed higher Infant Motor Activity Log parent ratings for I-ACQUIRE groups than usual treatment, supporting future analyses and next steps. Presented at the 2026 International Stroke Conference, I-ACQUIRE was a study that randomly assigned children aged 8-36 months with PAIS to 3 groups across 15 sites: usual care, moderate-dose I-ACQUIRE, and high-dose I-ACQUIRE. Those in the moderate dose group experienced treatment for 3 hrs/day, 5 days/week, over a 4-week span, while those on high-dose I-ACQUIRE were on the intervention for 6 hrs/day, 5 days/ week, for the same 4-week time period. Led by Warren D. Lo, MD, a clinical professor of pediatrics and neurology at Nationwide Children’s Hospital, the blinded study used the 30-item Emerging Behaviors Scale as the primary outcome, with favorable outcomes considered at least a 7-point gain at both post-treatment time points. Within the modified intent-to-treat group, consisting of 167 patients, results showed that at the end of treatment, both the moderate-dose I-ACQUIRE and high-dose I-ACQUIRE dosed groups outperformed usual treatment.

For more direct access to expert insight, head to NeurologyLive.com. Be sure to tune in next week to stay up to date on the latest in neurology. I’m Louie Pasculli thanks for watching Neurology News network.