Investigating Treatment Differences of Off-Label and Marketed Therapies for NMOSD: Sumaira Ahmed

WATCH TIME: 2 minutes

"We are super hopeful and excited about all that we will learn, especially what it will mean for patients, about the different therapies available to [those with NMOSD] and which ones may be best suited for them."

Despite the influx of recently approved therapies for patients with neuromyelitis optica spectrum disorder (NMOSD), patients still opt to use off-label treatments like rituximab to suppress disease activity. Over the years, global use of rituximab has been associated with reduced relapse rates in patients, although some studies have noted relapses linked to factors such as B-cell reconstitution or treatment gaps. Several in the field feel that comparing the efficacy of these off-label agents with other approved therapies such as satralizumab (Enspryng; Genentech), eculizumab (Soliris; Alexion), ravulizumab (Ultomiris; Alexion), and inebilizumab (Uplizna; Amgen), may ultimately help inform treatment strategies for patients with NMOSD.¹

Although each of these therapies has shown benefit in reducing relapse risk, there is limited direct comparative data to guide personalized treatment decisions. In recent news, The Sumaira Foundation (TSF), an advocacy organization dedicated to those living with NMOSD, announced a major international research initiative that will evaluate the head-to-head clinical efficacy of both approved and off-label options for NMOSD.⁴

At the 2025 Consortium of Multiple Sclerosis Centers (CMSC) Annual Meeting, held May 28-31, 2025, in Phoenix, Arizona, Sumaira Ahmed, founder and executive director of TSF, sat down with NeurologyLive^® to share this newly initiated study, funded with a $9 million PCORI award.⁵ In the interview, she noted that the upcoming trial will compare efficacy of both approved and commonly used off-label therapies for patients with NMOSD. Ahmed also highlighted that the study would gather real-world data, with the goal of generating evidence that will support clinical decision-making in managing NMOSD.

Click here for more coverage of CMSC 2025.

REFERENCES
1. Barreras P, Vasileiou ES, Filippatou AG, et al. Long-term Effectiveness and Safety of Rituximab in Neuromyelitis Optica Spectrum Disorder and MOG Antibody Disease. Neurology. 2022;99(22):e2504-e2516. doi:10.1212/WNL.0000000000201260
2. Wingerchuk DM, Fujihara K, Palace J, et al. Long-Term Safety and Efficacy of Eculizumab in Aquaporin-4 IgG-Positive NMOSD. Ann Neurol. 2021;89(6):1088-1098. doi:10.1002/ana.26049
3. Pittock SJ, Barnett M, Bennett JL, et al. Ravulizumab in Aquaporin-4-Positive Neuromyelitis Optica Spectrum Disorder. Ann Neurol. 2023;93(6):1053-1068. doi:10.1002/ana.26626
4. Traboulsee A, Greenberg BM, Bennett JL, et al. Efficacy and safety of satralizumab monotherapy for relapse prevention in neuromyelitis optica spectrum disorder (NMOSD): results from SAkuraStar, a double-blind placebo-controlled phase 3 clinical study. J Neurol Sci. 2019;405:116499. doi:10.1016/j.jns.2019.116499
5. PCORI. A Comparative Clinical Effectiveness Trial of Rituximab versus Ravulizumab, Inebilizumab, Satralizumab and Eculizumab To Prevent Relapses in Neuromyelitis Optica Spectrum Disorder. Accessed June 4, 2025. https://www.pcori.org/research-results/2025/immunotherapies-prevent-relapse-neuromyelitis-optica-spectrum-disorder