New SPINRAZA® (nusinersen) Data Reinforce Sustained Efficacy and Longer-Term Safety Across Broad Range of SMA Patients

Biogen Inc. recently announced additional data from the SPINRAZA (nusinersen) clinical development program that further demonstrate the sustained efficacy and longer-term safety of SPINRAZA in a broad range of patients with spinal muscular atrophy (SMA). These new data were selected for presentation at the 72nd American Academy of Neurology (AAN) annual meeting and will be available online via the 2020 AAN Science Highlights virtual platform.

The SHINE open-label extension study (NCT02594124) has enrolled 292 patients (infants through teenagers) from five previous SPINRAZA clinical studies, including ENDEAR. New findings from the SHINE study show treatment with SPINRAZA resulted in motor function improvement or disease stabilization in toddlers, children, and young adults who were treated continuously, some for up to 6 and a half years.

Key highlights include:

  • Patients with infantile-onset SMA included in the ENDEAR-SHINE study (n=105) and who had earlier initiation of SPINRAZA treatment experienced the greatest benefit, and those with later initiation showed evidence of motor function stabilization or improvement.
  • A separate analysis evaluated a cohort of seven young adults (Type 2 or 3) who began treatment with SPINRAZA as teenagers (aged 13 to nearly 16 years old) and have since been treated for up to 6 and a half years (range of 5.3 to 6.8 years). Most of these patients demonstrated generally stable or improved motor function throughout the follow-up period as assessed by the Hammersmith Functional Motor Scale Expanded (HFMSE), Revised Upper Limb Module and Upper Limb Module (RULM/ULM), and Six-Minute Walk Test (6MWT).
  • Results also measured the impact on participants’ caregivers via the Assessment of Caregiver Experience with Neuromuscular Disease (ACEND), with the majority reporting stable or decreased impact over the same period. ACEND is an outcomes instrument specifically designed to assess the caregiver impact experienced by caregivers raising children affected by neuromuscular disease, including physical, emotional and financial domains.
  • The durability of SPINRAZA was also demonstrated in individuals with later-onset SMA (n=126), as HFMSE and RULM scores were stable.
  • In all SHINE presentations, the safety profile of SPINRAZA was consistent with previously reported findings.