CMSC 2026: Real-World Analysis Supports Ocrelizumab Use in Pediatric-Onset Multiple Sclerosis

New real-world data presented at the 2026 Consortium of Multiple Sclerosis Centers (CMSC) Annual Meeting suggest that ocrelizumab (Ocrevus; Genentech) maintains similar effectiveness and safety in pediatric-onset multiple sclerosis (POMS) compared with young-adult onset disease, further supporting the expanding role of high-efficacy B-cell therapies in younger patients with relapsing MS.¹

The analysis, conducted using the international MSBase Registry, comes shortly after the FDA expanded ocrelizumab’s label in May to include pediatric patients aged 10 years and older with relapsing-remitting MS (RRMS), marking the first approval of the anti-CD20 therapy in children and adolescents with the disease.²

Pediatric-onset MS accounts for approximately 3% to 5% of all MS cases worldwide and is often characterized by higher inflammatory activity, greater relapse frequency, and substantial MRI lesion burden early in the disease course.^1,2 Investigators noted that although pediatric patients frequently recover well from early relapses, the cumulative inflammatory burden may contribute to long-term disability progression and cognitive impairment over time.

“These real-world findings suggest that the established efficacy and safety of ocrelizumab in young adults are generalizable to the pediatric-onset MS population,” Helmut Butzkueven, PhD, MBBS, The The Van Cleef Roet Chair of Neuroscience and Head of the Department of Neuroscience, Monash University, and colleagues, wrote.¹

In the matched cohort analysis, investigators compared 55 pediatric-onset patients aged 10 to younger than 18 years with 55 young-adult patients aged 18 to 30 years treated with ocrelizumab. Cohorts were matched using cardinality matching to control for sex, disease duration, baseline Expanded Disability Status Scale (EDSS) score, prior treatment exposure, MS phenotype, and country.

Across both groups, relapse outcomes were nearly identical. Pediatric and young-adult cohorts each experienced 8 relapses over 146.5 follow-up years, translating to an annualized relapse rate (ARR) of 0.055 (95% CI, 0.024-0.108; P = .999). Time to first relapse also did not significantly differ between groups (HR, 0.85; 95% CI, 0.29-2.54; P = .772). Similarly, there was no significant difference in time to first new or enlarging T2 lesion (HR, 1.40; 95% CI, 0.50-3.94; P = .522).¹

One of the more notable findings was the difference in disability improvement between cohorts. Pediatric patients demonstrated significantly greater confirmed disability improvement over follow-up, with a mean EDSS score change of −0.54 (SD, 1.19) compared with −0.03 (SD, 0.63) among young-adult patients (P = .002).

Safety outcomes were generally favorable and consistent across age groups. Adverse events were infrequent, occurring in 6 pediatric patients and 7 young-adult patients, and included asthma, constipation, COVID-19, fatigue, herpes zoster, migraine, and suicide attempts. No infections or malignancies were reported in either cohort.

The findings build on momentum generated by the phase 3 OPERETTA 2 trial (NCT05123703), which formed the basis of the recent FDA pediatric approval.² In that randomized, double-blind study of 187 pediatric patients with RRMS, ocrelizumab demonstrated noninferiority to fingolimod (Gilenya; Novartis) for annualized relapse rate while also showing superior MRI outcomes.

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Results from OPERETTA 2 showed a 48% relative reduction in ARR with ocrelizumab compared with fingolimod (rate ratio, 0.52; 95% CI, 0.19-1.33), the first FDA-approved treatment for pediatric MS. Other data from the ocrelizumab cohort within OPERETTA 2 revealed 48% fewer new or enlarging T2 lesions and 87% fewer gadolinium-enhancing T1 lesions at 12 weeks. Investigators additionally reported that no adverse events led to treatment withdrawal in the ocrelizumab arm.²

Ocrelizumab’s expansion into pediatric MS represented the latest milestone in the therapy’s evolution since its original FDA approval in 2017 for adults with relapsing forms of MS and primary progressive MS (PPMS), becoming the first approved treatment for PPMS at that time.³ Mechanistically, the anti-CD20 monoclonal antibody targets CD20-expressing B cells implicated in inflammatory demyelination and axonal injury in MS.

Over the past decade, ocrelizumab has become one of the most widely used high-efficacy therapies in MS, supported by long-term extension data demonstrating durable relapse suppression, MRI control, and slowing of disability progression.⁴ More recently, in 2024, the FDA approved a subcutaneous formulation, Ocrevus Zunovo, offering a shorter administration alternative to intravenous infusion for adults with relapsing MS and PPMS.⁵

Taken together, investigators said the latest MSBase findings provide additional reassurance that ocrelizumab’s efficacy and safety profile in adults appears reproducible in pediatric populations, an important consideration as clinicians increasingly move toward earlier use of high-efficacy therapies in younger patients with active disease.

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REFERENCES
1. Butzkueven H, Gershon A, Muros-Le Rouzic E, et al. Effectiveness and Safety of Ocrelizumab in People With Pediatric- vs Young-Adult Onset Multiple Sclerosis: An MSBase Registry Matched Cohort Analysis. Presented at: 2026 CMSC Annual Meeting; May 27-30, 2026; Charlotte, NC. Abstract NDM04.
2. Banwell B, Kotulska-Jozwiak K, Rostásy K, et al. Efficacy and safety of ocrelizumab compared with fingolimod in pediatric relapsing-remitting MS: results of the phase III OPERETTA 2 study. Presented at: ECTRIMS Congress; September 24-26, 2025; Barcelona, Spain. Late-Breaking Abstract O130. ECTRIMS Congress
3. Hauser SL, Bar-Or A, Comi G, et al. Ocrelizumab versus interferon beta-1a in relapsing multiple sclerosis. N Engl J Med. 2017;376(3):221-234. doi:10.1056/NEJMoa1601277. NEJM Article
4. Montalban X, Hauser SL, Kappos L, et al. Ocrelizumab versus placebo in primary progressive multiple sclerosis. N Engl J Med. 2017;376(3):209-220. doi:10.1056/NEJMoa1606468. NEJM Article
5. Genentech. FDA approves Ocrevus Zunovo (ocrelizumab and hyaluronidase-ocsq) for relapsing and progressive multiple sclerosis. News release. September 2024. Accessed May 28, 2026. https://www.roche.com/media/releases/med-cor-2024-09-16