Biogen presented Phase 3 study data on Spinraza (nusinersen) that showed a highly statistically significant and clinically meaningful improvement in motor function in children with later-onset spinal muscular atrophy (SMA) vs. untreated children. The findings were presented at the American Academy of Neurology (AAN) annual meeting in Boston, MA.

The CHERISH study was a multicenter, randomized, double-blind, sham-procedure controlled study to evaluate the safety and efficacy of Spinraza in children with later-onset SMA (n=126) aged 2–12 years. 

In the end-of-study analysis, children who took Spinraza had a highly statistically significant and clinically meaningful improvement in motor function with a treatment difference of 4.9 points in the mean change from baseline to Month 15 in the HFMSE score (P=0.0000001). Compared to baseline, children who took Spinraza had a 3.9 point average improvement at Month 15, while untreated children had a mean decline of 1.0 point. 

The primary endpoint data at the end-of-study analysis was consistent with the data seen at the interim analysis. Other endpoints, including achieving new motor milestones and upper limb motor function, were also consistently in favor of pediatric patients who received treatment. Moreover, the frequency of treatment-related adverse events (AEs), severe AEs and serious AEs (SAEs) were all less in Spinraza-treated patients vs. untreated patients.  

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Interim data from the Phase 2 NURTURE study will also be presented at AAN, which evaluated Spinraza for infants <6 weeks old with genetically diagnosed SMA who were presymptomatic at treatment initiation. At the time of analysis, 20 infants were enrolled and all were alive and none required respiratory intervention. Most infants attained motor milestones and growth parameter gains generally consistent with normal development as measured by validated scales.

Alfred Sandrock, MD, PhD, executive vice president and chief medical officer at Biogen, added, ““The results from NURTURE are significant, as they continue to demonstrate the importance of beginning Spinraza treatment as soon as possible after an SMA diagnosis and the major impact that early treatment may have across a broad range of SMA populations.”

Spinraza, an antisense olignucloetide, was approved by the Food and Drug Administration (FDA) in December 2016. It is available as 12mg/5mL strength solution for intrathecal injection in single-dose vials.

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