Spinraza vs Zolgensma

Spinraza (nusinersen) is an antisense oligonucleotide therapy reviewed for spinal muscular atrophy (SMA). Its review documents highlight improvements in motor milestone development and CHOP INTEND scores in treated patients compared to control groups. Zolgensma (onasemnogene abeparvovec) is a one-time gene therapy indicated for patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene. Its review documents include data on survival in mouse models and changes in peroneal CMAP amplitude in human trials.

The pivotal trials for each drug were conducted independently with distinct designs, patient populations, and timepoints. Spinraza's review noted that 40% of treated patients showed improvement in motor milestone development, compared to 0% in the control group. Zolgensma's review included data such as a median survival of 282 days for treated SMNΔ7 mice (ICV, highest dose) versus 17.5 days for untreated mice. The trials used different endpoints, such as motor milestone development and CHOP INTEND scores for Spinraza, and peroneal CMAP amplitude and survival in animal models for Zolgensma. The patient populations and the duration of follow-up also varied between the studies. It is crucial to understand that these figures are not from head-to-head comparisons, and neither drug is presented as superior based on these separate trial results.