ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated with Onasemnogene Abeparvovec-brve


Topic:

Clinical Trials

Poster Number: 88 S

Author(s):

Bakri Elsheikh, MBBS, The Ohio State University Wexner Medical Center, Nayla Mumneh, MD, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, United States, Elizabeth Nguyen, PharmD, MBA, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, United States, Andreja Ilic, MA, Novartis Pharmaceuticals

Background: Onasemnogene abeparvovec-brve (OA-brve) was FDA-approved in November 2025 as an intrathecal (IT) adeno-associated-virus (AAV9)–mediated gene-replacement therapy in spinal muscular atrophy (SMA) patients ≥2 years of age. Approval was based on proven safety, efficacy, and tolerability of OA-brve in the global phase III STEER study that included non-independently ambulant SMA patients between 2 and <18 years of age. Objectives: STREAM is a US multicenter, single-arm, pragmatic study to address critical evidence needs in the treatment landscape for patients ≥ 2 years of age with SMA with focus on the on-label population of OA-brve. Methods: STREAM aims to enroll 36 patients. Consented patients will be enrolled in 3 predefined treatment cohorts: 1) independently ambulatory patients ages 2 to < 18, 2) independently ambulatory patients ages ≥18 years), and 3) non-independently ambulatory patients who are ≥18 years and have a ≥1 baseline entry level of the revised upper limb module (RULM). Each patient will receive the single dose of OA-brve: 1.2 × 10¹⁴ vector genomes delivered in a 3-mL bolus via lumbar puncture. The study will be conducted with a post-treatment follow-up of biannual in-clinic visits for 24 months followed by remote annual visits up to 3 years. The study will measure and characterize longitudinal motor-function trajectories utilizing the Hammersmith Functional Motor Scale (HFMSE) and/or RULM through 24 months; evaluate adverse events (AEs), serious AEs, and clinical laboratory assessments during safety monitoring; characterize longitudinal trajectories of patient-reported outcomes through 24 months, 3 years via annual remote follow-up, and describe healthcare-resource utilization and supportive-care needs to contextualize the therapy’s impact on real-world clinical management. Conclusions: Evidence generated in this study will bridge the gap between a broader US label and earlier trial populations, support clinician decision-making, inform payer evaluations of economic value, and generate long-term data on patients treated with OA-brve.