Spinraza vs Evrysdi

Spinraza (nusinersen) is an antisense oligonucleotide injection reviewed for spinal muscular atrophy (SMA). Regulators noted an improvement in motor milestone development in 40% of patients in its pivotal trial, compared to 0% in the control group. Evrysdi (risdiplam) is an oral treatment for 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. In its pivotal trial, 90.2% of patients achieved an increase of at least 4 points in the CHOP-INTEND score from baseline.

These drugs were studied in separate trials with different designs, patient populations, and timepoints. The endpoints, comparator arms, and specific measures of efficacy varied between the studies. For example, Spinraza's review highlighted motor milestone development and CHOP INTEND score improvement, while Evrysdi's review noted SMN protein increase and the proportion of patients sitting without support. The figures presented for each drug are from their respective regulatory reviews and should not be compared directly as if from a single trial. Neither drug is presented as superior to the other based on these separate findings.