EU label for Spinraza updated in Spinal Muscular Atrophy

Biogen announced an update to the European label for Spinraza (nusinersen), incorporating longer-term data from four studies (NURTURE, EMBRACE, SHINE and CS3A) which includes longer-term data across a broad range of ages and types of Spinal Muscular Atrophy. These studies, which looked at pre-symptomatic, infantile-onset and later-onset treatment, further confirm the benefits of early treatment and durability of treatment effect of nusinersen. No new safety concerns were identified.

Collectively, these data demonstrate that treatment with nusinersen results in continuous improvement or stabilization of disease activity for patients of all ages. Whilst the benefits are greatest with earlier treatment initiation, the SHINE data, which followed patients from the pivotal CHERISH and ENDEAR studies, also demonstrates that patients starting treatment at a later stage can still gain significant improvements in motor function and abilities that can help them in their day-to-day lives. At the time of interim analysis of SHINE, length of treatment with nusinersen was up to 474 days for patients enrolled from CHERISH and up to 592 days for those from ENDEAR.

The European label update also includes data from EMBRACE which examined both infantile and later-onset SMA, final results from the CS3A study (which investigated the use of nusinersen in symptomatic patients with infantile-onset SMA) as well as data from the NURTURE study. NURTURE is an ongoing, open-label study of pre-symptomatic infants genetically diagnosed with SMA, the latest results of which are being presented at the CureSMA and EAN Congresses in the coming days.