Project Lead: Claudia Wild
Author: Claudia Wild
Publication: March 2020
Language: German
In June 2017, Nusinersen (Spinraza®) was approved broadly – for all SMA Typologies (despite lack of evidence) – by the European Medicines Agency (EMA) for the treatment of chromosome 5q13 (5q)-associated SMA. The question of the update presented here is which scientific evidence from clinical intervention or observational studies for the medium (or long-term) benefit of Nusinersen (Spinraza®) in children and adolescents > 6 years with SMA "late onset" (SMA 2 + 3) is available. Three publications on the question (SMA 2 + 3 with a total of 40 patients) were identified. The available evidence for medium-term (> 12 months) follow-up data comes from a single prospective sponsor-funded ISIS CS12 study, as well as from a small retrospective study (of poor quality). Even after more than two years after the approval of Nusinersen, the evidence for this patient population is of extremely low quality, lacking validity.