Biogen's Salanersen Shows Promise for SMA Patients Post-Gene Therapy

Biogen’s experimental drug, Salanersen, is showing promising potential for treating children with spinal muscular atrophy (SMA), even after they’ve received other gene therapies like Zolgensma. Unlike Spinraza, which requires multiple spinal taps, Salanersen could become a more convenient, once-a-year treatment. In early trials, the drug has demonstrated substantial slowing of neurodegeneration and helped children achieve key developmental milestones, such as sitting unsupported, crawling, and standing—milestones that were previously out of reach for these patients.

In the first phase of testing, children between 6 months and 12 years old received Salanersen at least six months after being treated with Novartis' Zolgensma. After a year, all the patients showed significant improvements.

Neurofilament levels, which are markers of nerve damage, were reduced by 70% after just six months of treatment, suggesting that Salanersen could not only slow disease progression but also improve physical function over time.

Given these results, Biogen is advancing Salanersen directly to Phase 3 trials, bypassing midstage testing. This decision highlights the company's confidence in the drug's potential to make a meaningful difference for children with SMA. The upcoming Phase 3 trials will explore the drug’s effectiveness in both previously treated and untreated patients, which could broaden its impact and further demonstrate its benefits.

Despite challenges in the stock market and stiff competition in the gene therapy space, Salanersen represents a breakthrough for SMA treatment. If successful, it could provide significant relief for families dealing with this devastating condition, offering hope for improved quality of life and better treatment options in the future.

As Biogen moves forward with Phase 3 testing, Salanersen stands out as a beacon of hope for SMA patients. If it continues to show promising results, it could change the landscape of SMA treatment, offering children and their families a much-needed alternative to current therapies.

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Created: June 26th, 2025

Citations:

Gatlin, A. (2025, June 25). Can Biogen save even more kids with this new drug? Investor's Business Daily. https://www.investors.com/news/technology/biogen-next-generation-spinraza-spinal-muscular-atrophy-treatment/?utm_source=chatgpt.com