Nayzilam (midazolam intranasal) / Sawai Pharma, UCB - LARVOL DELTA

Healthcare Resource Utilization and Costs With the Introduction of Intranasal Midazolam in Acute Seizure Management: A Wisconsin-Based Claims Analysis (AES 2025) - "HCRU and costs were reduced following introduction of MDZ nasal spray. Increases in mean pharmacy costs were offset by reduced inpatient, ED, and outpatient visits and reduced total costs. While not assessed in this analysis, quality of life improvements may be experienced with these reduced inpatient and ED visits."

HEOR • Late-breaking abstract • CNS Disorders • Epilepsy

Describing the Population of Patients With Prolonged Seizures: US Subgroup Results From a Global Real-World Point-In-Time Study (AES 2025) - "70% of PwE with PS were currently prescribed RM, and oral benzodiazepines (including buccal midazolam) were the most commonly prescribed RM (oral benzodiazepines: 37% [n=141/379]; intranasal midazolam: 32% [n=122/379]; intranasal diazepam: 27% [n=102/379]; rectal diazepam: 7% [n=25/379])... PwE experiencing PS are reported to encounter progression to status epilepticus and/or seizure clusters, leading to emergency care, and hospital, emergency room, and intensive care unit admissions. Approximately two-thirds of PwE with PS have a seizure action plan for PS. PS have a subsequent impact on seizure worry among some PwE with PS."

Clinical • Real-world • Real-world evidence • CNS Disorders • Epilepsy

A mismatch in enzyme-redox partnerships underlies divergent cytochrome P450 activities between human hepatocytes and microsomes. (PubMed, Commun Biol) - "Mechanistically, we serendipitously discover that HH CYP3A4-mediated midazolam-metabolism is increased by gefitinib and find that this can only be recapitulated in non-canonical Cytb5R-dependent NADH-HLM and recombinant CYP3A4-Cytb5R. We conclude that Cytb5R is important for HH-CYP3A4 and show HH-consistent CYP3A4-activities in NADH-HLM. Imaging NADH/NADPH in hepatocytes shows equivalent concentrations suggesting CYP redox-partnerships are cofactor-independent and likely influenced by protein-protein interactions as mimicking the dense-intracellular protein using albumin recapitulates HH savolitinib-metabolism in HLM."

Journal • Licensing / partnership • CYB5A • CYP1A2 • CYP3A4

A Study to Assess the Time to Onset of Action of Staccato Alprazolam Versus Midazolam and Diazepam in Healthy Participants (clinicaltrials.gov) - P1 | N=50 | Recruiting | Sponsor: UCB Biopharma SRL | Not yet recruiting ➔ Recruiting

Enrollment open • CNS Disorders

A Study to Assess Drug-drug Interaction of ZX008 in Healthy Male and Female Study Participants (clinicaltrials.gov) - P1 | N=22 | Completed | Sponsor: UCB BIOSCIENCES, Inc. | Active, not recruiting ➔ Completed

Trial completion

A Study to Assess the Time to Onset of Action of Staccato Alprazolam Versus Midazolam and Diazepam in Healthy Participants (clinicaltrials.gov) - P1 | N=50 | Not yet recruiting | Sponsor: UCB Biopharma SRL

New P1 trial • CNS Disorders

Medications used for seizure-emergency management in the UK community: A clinical practice research datalink retrospective database study. (PubMed, Epilepsia Open) - "In people with epilepsy, some seizures last too long and will not stop on their own, or may happen one after another, becoming emergencies that need medical attention; seizure-emergency medications are given to stop the seizure and prevent other medical problems. We looked at the share of people with epilepsy who had a seizure-emergency medication prescription in a UK database, and this group's use of health services. In 2019, 7% of people with epilepsy were prescribed a seizure-emergency medication. Use of health services was high in this group, highlighting the need for better treatment options."

Journal • Retrospective data • CNS Disorders • Epilepsy

A Study to Assess Drug-drug Interaction of ZX008 in Healthy Male and Female Study Participants (clinicaltrials.gov) - P1 | N=22 | Active, not recruiting | Sponsor: UCB BIOSCIENCES, Inc. | Recruiting ➔ Active, not recruiting

Enrollment closed

A Study to Assess Drug-drug Interaction of ZX008 in Healthy Male and Female Study Participants (clinicaltrials.gov) - P1 | N=22 | Recruiting | Sponsor: UCB BIOSCIENCES, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open

Seizure Clusters and Rescue Medication: What Does Youtube Offer? (AES 2024) - "FDA-approved treatment recommendations included rectal Diastat, intranasal Nayzilam, or intranasal Valtoco... Finding informative YouTube videos on seizure cluster management is difficult. While several videos appeared to be effective as patient and provider education, applying less specific terminology returned unrelated or unhelpful videos. Healthcare institutions and pharmaceutical companies should produce brief, helpful videos of rescue therapy and arm patients with search terms to facilitate better navigation of educational resources."

CNS Disorders • Epilepsy

Comprehensive Analysis of Lennox-gastaut Syndrome in Europe: Treatment Patterns, Healthcare Utilisation, and Quality of Life (AES 2024) - "The most prescribed ASMs were valproate, clobazam, lamotrigine, rufinamide, levetiracetam and cannabidiol...The most prescribed RMs were diazepam, midazolam... Despite receiving multiple treatment regimens, and 70% being prescribed RMs, patients with LGS continue to experience GTC and drop seizures into adulthood. Hospitalisations persist for 16%, most of whom are admitted via ER and one-fifth to the ICU. Almost half of patients' QoL is rated as "poor" by their physicians."

HEOR • CNS Disorders • Epilepsy • Pediatrics

A Study to Assess Drug-drug Interaction of ZX008 in Healthy Male and Female Study Participants (clinicaltrials.gov) - P1 | N=22 | Not yet recruiting | Sponsor: UCB BIOSCIENCES, Inc.

New P1 trial

Return to full baseline functionality after repeated intermittent use of midazolam nasal spray in patients with seizure clusters: Post hoc analysis of an open-label extension trial. (PubMed, Epilepsy Behav) - P3 | "In almost all patients, RTFBF was observed within 24 h of MDZ-NS administration. The median time to RTFBF was similar in SCEs treated with one or two doses, and stable over the course of repeated intermittent use. Thus, dose (5 or 10 mg) does not seem to be a key influencer of time for RTFBF after repeated intermittent use."

Journal • Retrospective data • CNS Disorders • Epilepsy

VAGAL NERVE STIMULATOR-INDUCED SLEEP DISORDERED BREATHING IN A PATIENT WITH REFRACTORY SEIZURES (CHEST 2024) - "Medications included Vimpat, Fycoma, Xcopri. Clonazepam was as needed and Nayzilam to be used as rescue if needed... This case report elucidates several novel aspects, informed by the provided data. VNS benefits in patients with refractory seizures but careful observation and monitoring is recommended with any patients with preexisting sleep disordered breathing."

Clinical • CNS Disorders • Epilepsy • Obstructive Sleep Apnea • Pain • Respiratory Diseases • Sleep Apnea • Sleep Disorder

Overview of preclinical and clinical studies investigating pharmacokinetics and drug-drug interactions of padsevonil. (PubMed, Expert Opin Drug Metab Toxicol) - P1 | "A study in genotyped participants confirmed the role of CYP2C19 in clearance and time-dependent kinetics; the major contribution of CYP3A4 was confirmed in DDI studies with CYP3A4-inducers (carbamazepine, oxcarbazepine) and -inhibitor (erythromycin). Padsevonil did not affect pharmacokinetics of valproate/lamotrigine/levetiracetam/oxcarbazepine or oral contraceptives. In a cocktail clinical study, padsevonil showed moderate CYP2C19 inhibition (omeprazole) and weak CYP3A4 induction (oral midazolam). No specific effects on CYP1A2 (caffeine), CYP2C9 (S-warfarin), and CYP2D6 (dextromethorphan) were observed. The studies presented helped in understanding padsevonil disposition and risks of DDIs, which would inform dosing and prescribing. https://www.clinicaltrials.gov/identifiers are NCT04131517, NCT03480243, NCT03695094, NCT04075409."

Journal • PK/PD data • Preclinical • CNS Disorders • Epilepsy • CYP1A2 • CYP2C19 • CYP2C9 • CYP3A4 • SLC22A1

Real-world use and associated health care resource utilization among patients with epilepsy receiving rescue midazolam nasal spray (AMCP 2024) - "Increased epilepsy-related HCRU in the month before MDZ-NS use suggests a precipitating event preceding the need for MDZ-NS. Compared with PwE who received 1 MDZ-NS, those who received 2 or more MDZ- NS fills may have had more severe epilepsy, as shown by a greater likelihood of having more than 2 prior/concomitant ASMs and higher epilepsy-related HCRU and costs. Overall, MDZ-NS was a minor contributor to pharmacy costs."

Clinical • HEOR • Real-world • Real-world evidence • Age-related Macular Degeneration • CNS Disorders • Epilepsy • Macular Degeneration • Ophthalmology • Retinal Disorders

Use of Benzodiazepines for Seizure Rescue in Adult Epilepsy Clinic Patients (AES 2023) - "Currently, there are only three US marketed, FDA approved seizure rescue medications: rectal diazepam (Diastat), intranasal midazolam (Nayzilam), and intranasal diazepam (Valtoco)...Half of these prescriptions were for unapproved seizure rescue medications, most frequently lorazepam (oral or sublingual) and clonazepam (oral)... BDZ for seizure rescue are widely used in the adult epilepsy population, often originally prescribed by providers outside the epilepsy clinic. Non-FDA approved rescue medications are often prescribed, and prescribing directions frequently are without specific instruction of when and how often to use. Documentation of use and lack of use is often lacking in the electronic medical record."

Clinical • CNS Disorders • Epilepsy

Application Of Plan-Do-Study-Act Method In Seizure Rescue Medication Practices: A Single Pediatric Neurology Clinic Quality Improvement Study (AAN 2023) - "Clonazepam ODT was the most prescribed rescue medication in both cohorts. There was an increase in prescribing trend of Valtoco and Nayzilam noted in the PDSA2 cohort...Conclusions Our study showed improvement of seizure rescue medication prescription practices in PDSA2 compared to PDSA1 cycles. The role of educational session for fellows, attending physicians and patient family regarding the timing of rescue medication, appropriate options, and correct dosing after the PDSA1 cycle was significant.Effective strategy of implementing systematic and rigorous standards in PDSA cycles sequentially could improve the prescription rate to 100% for convulsive seizures and consequently result in reduction in number of ED visits for breakthrough seizures and status epilepticus admissions."

Clinical • CNS Disorders • Epilepsy • Pediatrics

Baseline Characteristics and Antiseizure Medication Use of Patients with Epilepsy in the Year Prior to Initiating Midazolam Nasal Spray, Diazepam Rectal Gel, or Diazepam Nasal Spray (AES 2022) - "Levetiracetam was the most common prior and concomitant ASM. New users of MDZ-NS and DZP-NS showed generally similar baseline characteristics, but were older with more prior/concomitant ASMs vs the DZP-RG cohort. Baseline characteristics and prior/concomitant ASM use were influenced by approved patient age and BZP prescribing information, with adults most commonly receiving MDZ-NS."

Clinical • Atrial Fibrillation • Cardiovascular • CNS Disorders • Epilepsy • Mood Disorders • Pain • Psychiatry

UCB to present data at the American Epilepsy Society 76th Annual Meeting 2022 supporting a decades-long commitment to transforming epilepsy care (PRNewswire) - "21 scientific presentations, including 7 late breakers, showcase the breadth of UCB's portfolio, reinforcing commitment to improving the lives of people living with epilepsy....UCB...today announced that data from its anti-seizure medication portfolio (brivaracetam, fenfluramine, lacosamide, and midazolam nasal spray) will be presented at the 76th American Epilepsy Society (AES) Annual Meeting (Nashville, Tennessee), December 2-6."

Clinical • Late-breaking abstract • Retrospective data • CNS Disorders • Epilepsy

Psychosocial outcomes of repeated treatment of seizure clusters with midazolam nasal spray: Results of a phase 3, open-label extension trial. (PubMed, Epilepsy Behav) - P3 | "Patients and caregivers perceived MDZ-NS favorably, with improvement from baseline on perceived effectiveness, side effects, convenience, and global satisfaction in the TSQM. This is supported by progressively lower anxiety and higher confidence levels about traveling with MDZ-NS over repeated intermittent use in the ITIQ. The positive mean changes observed in SF-12v2 scores from baseline to the last visit were small in magnitude. Limitations of this exploratory analysis include the open-label trial design and that these questionnaires have not been directly validated in epilepsy to identify clinically important changes; however, this does not mean these findings are not clinically meaningful. Overall, MDZ-NS is a socially acceptable drug device for outpatient treatment of seizure clusters that has the potential to improve quality of life and overall independence."

Journal • P3 data • CNS Disorders • Epilepsy • Mood Disorders • Pain • Psychiatry

Midazolam nasal spray to treat intermittent, stereotypic episodes of frequent seizure activity: pharmacology and clinical role, a comprehensive review. (PubMed, Health Psychol Res) - "Many studies have indicated quick cessation of seizures with Nayzilam compared to rectal diazepam, which has been the standard of care treatment. Nayzilam has been proven to be safe and effective for acute seizures in children, deeming it a revolutionary alternative in times where intravenous administration is not possible."

Journal • Review • CNS Disorders • Epilepsy

BioCareSD Adds Six New Products to Existing Portfolio in Partnership with UCB (BioSpace) - "BioCareSD...announced a contract with UCB to add six new product offerings to its already diverse portfolio. This partnership, which will enable BioCareSD to service a wider patient population, demonstrates the organization’s continued commitment to being the industry’s most trusted healthcare partner....BRIVIACT® - partial-onset seizures in patients one month of age and older CIMZIA® - Crohn’s Disease and Rheumatoid Arthritis NAYZILAM® - intermittent, stereotypic episodes of frequent seizure activity, distinct from patient’s usual seizure pattern in patients with epilepsy 12 years of age and older NEUPRO® - Parkinson’s Disease and Restless Leg Syndrome KEPPRA® - partial-onset seizures in adults with epilepsy VIMPAT® - partial-onset seizures in patients with epilepsy 17 years of age and older."

Licensing / partnership • CNS Disorders • Crohn's disease • Epilepsy • Parkinson's Disease • Restless Legs Syndrome • Rheumatoid Arthritis

Strong first six months – UCB with continued delivery and strong resilience (UCB Press Release) - "VIMPAT® (lacosamide) for people living with epilepsy showed net sales of € 744 million....KEPPRA® (levetiracetam) available for people living with epilepsy reported 22% lower net sales....BRIVIACT® (brivaracetam), used by people living with epilepsy showed continued, significant growth in all regions Briviact® is available to patients....NAYZILAM® (midazolam) Nasal SprayCIV, a nasal rescue treatment for epilepsy seizure clusters in the U.S. (launched since December 2019) reached net sales of € 36 million after € 21 million. FINTEPLA® (fenfluramine) is now part of the UCB epilepsy portfolio thanks to the completed acquisition of Zogenix, Inc. in early March....Net sales (March - June) were € 35 million."

Commercial • Sales • CNS Disorders • Epilepsy

UCB Showcases Strength of Broad Neurology Portfolio at 74th American Academy of Neurology (AAN) Annual Meeting (PRNewswire) - "Epilepsy...UCB will be reporting data from several significant clinical epilepsy studies from its broad epilepsy portfolio. Notably, these data include real-world, retrospective, interim evidence evaluating the effectiveness and tolerability of brivaracetam for adult patients with partial-onset seizures...Additionally, presented data will evaluate dose-response relationships for the impact of fenfluramine on everyday executive function in patients, as captured using the Behavior Rating Inventory of Executive Function (BRIEF®)....Data will also summarize the early use of midazolam nasal spray in patients with seizure clusters."

Clinical data • Retrospective data • CNS Disorders • Epilepsy