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Nya data för Spinraza bekräftar bibehållen effekt och säkerhet över tid för ett brett spektrum av patienter med spinal muskel atrofi

Nya data för Spinraza® (nusinersen) bekräftar bibehållen effekt och säkerhet över tid för ett brett spektrum av patienter med spinal muskel atrofi (SMA)

  • Behandling med Spinraza förbättrade eller stabiliserade motoriska funktioner hos olika typer av patienter, inklusive unga vuxna.
  • Spinrazas långsiktiga säkerhetsprofil var densamma för ett brett spektrum av åldrar och typer av SMA.
  • Nya data bidrar till den betydande mängden evidens som bekräftar den kliniskt meningsfulla och kvarstående nyttan som Spinraza ger hos småbarn, barn och unga vuxna behandlade i upp till sex och ett halvt år.

Cambridge, Mass. – May 18, 2020 – Biogen Inc. (Nasdaq: BIIB) today 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.

“As the first approved treatment for SMA, SPINRAZA offers a significant data set that allows us to assess the safety and durability of repeated doses over time in individuals across age groups and varying disease severity,” said Alfred Sandrock, Jr., M.D., Ph.D., Executive Vice President, Research and Development at Biogen. “New data show that continuous treatment with SPINRAZA for up to six and a half years improved or stabilized motor function and disease activity in a broad spectrum of patients with SMA. These results are in stark contrast to the expected natural history of the disease. Further, in a progressive disease like SMA, stabilization is an important measure of treatment success, allowing patients to retain motor function that may otherwise be lost.”

New Data Reinforce Sustained Efficacy and Longer-Term Safety of SPINRAZA Across Age Groups and SMA Types

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 six 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 six 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.1
  • 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.

AAN data presentations highlighted in this release include:

  • Nusinersen in Infantile-onset Spinal Muscular Atrophy: Results From Longer-term Treatment From the Open-label SHINE Extension Study
  • Longer-term Experience With Nusinersen in Young Adults With Spinal Muscular Atrophy: Results From the CS2/CS12 and SHINE Studies
  • Longer-term Treatment With Nusinersen: Results in Later-onset Spinal Muscular Atrophy From the SHINE Study
  • Safety Profile of Nusinersen in Presymptomatic and Infantile-Onset Spinal Muscular Atrophy (SMA): Interim Results From the NURTURE and ENDEAR-SHINE Studies

Om Spinraza

Spinraza är avsett för behandling av spinal muskelatrofi av typ 5q. Biverkningar som observerats är fall av allvarlig infektion, såsom meningit. Det har även förekommit rapporter om kommunicerande hydrocefalus, aseptisk meningit och överkänslighet (t.ex. angioödem, urtikaria och hudutslag). För information om kontraindikationer, varningar och försiktighet, biverkningar, dosering och förpackningar se


  1. Matsumoto H, Clayton-Krasinski DA, Klinge SA, et al. Development and initial validation of the assessment of caregiver experience with neuromuscular disease. J Pediatr Orthop. 2011;31(3):284-292.
  2. Based on Commercial Patients, Early Access Patients, and Clinical Trial Participants as of March 31, 2020.
  3. Finkel R, Chiriboga C, Vajsar J, et al. Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study. Lancet. 2016;388(10063):3017-3026.
  4. Darras B, Markowitz J, Monani U, De Vivo D. Chapter 8 – Spinal Muscular Atrophies. In: Vivo BTD, ed. Neuromuscular Disorders of Infancy, Childhood, and Adolescence (Second Edition). San Diego: Academic Press; 2015:117-145.

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Spinraza kan ha potentiella fördelar efter genterapi hos spädbarn och barn med SMA