Biogen announced that the U.S. Food and Drug Administration approved a higher‑dose regimen of its spinal muscular atrophy therapy, Spinraza (nusinersen), on March 30 2026. The new dosing schedule calls for 50 mg/5 mL loading doses followed by 28 mg/5 mL maintenance doses, a change designed to deliver a higher concentration of drug during both the loading and maintenance phases and to mitigate the waning‑off effect observed with the standard 12 mg schedule.
The approval follows a prior FDA complete‑response letter issued in September 2025 that cited manufacturing concerns. Biogen resolved those issues, and the agency’s March 30 decision confirms that the high‑dose regimen meets all safety and efficacy requirements. The DEVOTE Phase 2/3 study provided the data, showing statistically significant improvements in motor function for treatment‑naïve infants compared with a sham group from the ENDEAR study, with a mean difference of 26.19 points on the CHOP‑INTEND scale (15.1 vs. –11.1). The high‑dose regimen also produced a more rapid reduction in neurofilament light chain levels, a biomarker of neurodegeneration.
From a business perspective, the approval offers Biogen a way to stabilize and potentially grow Spinraza sales after a 15 % year‑over‑year decline in revenue in Q4 2025 and a 2 % decline for the full year. The new regimen positions the company to better compete with Roche’s Evrysdi and Novartis’ Zolgensma, both of which have captured market share in the SMA space. By expanding the dosing range, Biogen can address unmet needs in both adult and pediatric patients and potentially broaden its addressable market while maintaining its rare‑disease franchise’s revenue base.
Management highlighted the clinical and commercial significance of the approval. Dr. Richard Finkel, director of the Center for Experimental Neurotherapeutics at St. Jude Children’s Research Hospital, said, “Optimizing the dose of nusinersen builds on a therapy that we already know can change lives. The high‑dose regimen demonstrated meaningful clinical benefit while maintaining a well‑characterized safety profile.” CEO Chris Viehbacher echoed this sentiment, noting that the approval could “reinvigorate Biogen’s SMA franchise” and serve as a “bridge to growth” toward next‑generation therapies such as salanersen.
Analysts at BMO Capital Markets and Jefferies welcomed the decision, describing it as a “meaningful first step in continuing to stabilize and grow the company’s SMA products.” They emphasized that the high‑dose approval could help sustain sales and provide a platform for future pipeline assets, reinforcing Biogen’s strategy to maintain its rare‑disease portfolio while advancing next‑generation treatments.
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