Biogen and Ionis have announced positive results from the Phase 3 study of Spinraza (nusinersen), an investigational treatment for spinal muscular atrophy(SMA) in children. The study, CHERISH, met its primary endpoint at the interim analysis by demonstrating statistically significant improvement in motor function compared to the non-treated group.

The 15-month study included a total of 126 non-ambulatory patients with later-onset SMA (consistent with Type 2). The participants had signs and symptoms at ≥6 months and were 2–12 years of age at screening. 

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From baseline to 15 months of treatment, patients who received Spinraza achieved a mean improvement of 4.0 points as measured by the Hammersmith Functional Motor Scale Expanded (HFMSE), while patients who were not on treatment declined by a mean of 1.9 points. A change of ≥3 points on HFMSE has been identified as clinically meaningful.

The analysis also displayed a favorable safety profile with no participants discontinuing. The CHERISH study will be halted and patients will be able to transition into the SHINE study, an open-label extension study intended to evaluate Spinraza’s long-term safety and tolerability.

Spinraza is an antisense oligonucleotide (ASO) that is designed to alter the splicing of the SMN2 gene in order to increase production of fully functional SMN protein. The Food and Drug Administration (FDA) accepted an New Drug Application (NDA) for Spinraza for SMA and is planning an expedited review of the treatment. 

Biogen is anticipating a potential launch of Spinraza at the end of 2016 or the first quarter of 2017. 

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