Spinraza, Zolgensma, and Evrysdi: Here’s What Every SMA Family Should Know
Three treatments that transformed the SMA landscape are Spinraza, Zolgensma, and Evrysdi, but each approaches the disease differently. Spinraza, also known as nusinersen, targets the backup SMN2 gene and helps it produce more functional SMN protein; it is approved for pediatric and adult patients and is administered into the cerebrospinal fluid through an intrathecal injection. Zolgensma, also known as onasemnogene abeparvovec-xioi, is a one-time intravenous gene replacement therapy approved in the United States for children under 2 years old with SMA caused by bi-allelic SMN1 mutations.
Evrysdi, also known as risdiplam, provides another approach by modifying SMN2 splicing so the body can make more full-length SMN protein. Unlike Spinraza’s spinal administration or Zolgensma’s one-time gene therapy infusion, Evrysdi is taken once daily and is available as an oral solution or, for eligible patients receiving the 5 mg dose, a tablet. The current FDA prescribing information indicates Evrysdi for pediatric and adult patients with SMA, including infants, with dosing determined by age and body weight. Every SMA family should understand that these treatments are not interchangeable simply because they address the same disease. Differences in mechanism, age eligibility, administration, dosing schedules, safety monitoring, medical history, spine anatomy, and previous treatment can all influence which therapeutic approach may be appropriate.
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