Biogen received a positive opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) on November 17, 2025, recommending approval of a high‑dose nusinersen regimen for 5q spinal muscular atrophy (SMA). The new protocol calls for two 50 mg intrathecal injections 14 days apart, followed by a 28 mg maintenance dose every four months, a change that builds on the safety and efficacy data from the DEVOTE study.
The DEVOTE study demonstrated a 68 % reduction in the risk of death or permanent ventilation and significant motor function gains in infants, supporting the clinical value of the higher dose. If approved, the regimen could broaden the patient population that can benefit from SPINRAZA and improve adherence by reducing the frequency of maintenance injections.
Biogen’s recent financials show that SPINRAZA generated $1.19 billion in worldwide sales in the first nine months of 2025, while the company’s total revenue for Q4 2024 was $2.5 billion, a 3 % year‑over‑year increase. The rare‑disease portfolio grew 13 % to $535 million, whereas the multiple‑sclerosis segment declined 8 % to $1.07 billion. The high‑dose approval could add incremental revenue to the rare‑disease segment and reinforce Biogen’s leadership in SMA therapy.
The U.S. regulatory path remains uncertain, as the FDA issued a Complete Response Letter on September 24, 2025, requesting updates to the Chemistry, Manufacturing, and Controls module for the higher dose. Biogen plans to resubmit, but the letter indicates that approval in the United States could be delayed beyond the anticipated April 3, 2026 decision, potentially limiting the timing of revenue gains in the largest market.
Priya Singhal, Executive Vice President and Head of Development, emphasized the urgency of addressing unmet needs in the SMA community, noting that the CHMP opinion “represents a promising advancement in our commitment to support evolving patient needs and deliver therapies that can enhance outcomes.” She added that Biogen is focused on expanding access while maintaining rigorous safety standards.
Analysts view the CHMP endorsement as a positive step that could strengthen Biogen’s competitive position in the SMA market. While the U.S. approval timeline remains a headwind, the European endorsement is expected to accelerate market penetration and could translate into additional revenue streams for the company’s rare‑disease portfolio.
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