Risdiplam (Evrysdi®) may benefit patients with spinal muscular atrophy (SMA) in a unique manner compared to other therapeutic options, according to a study recently published in the journal Expert Opinion on Orphan Drugs.

“Nevertheless, having an option to choose the most effective and most tolerated drug on an individual basis is a big step forward in the treatment of SMA both for patients and treating physicians,” wrote Nicolas Deconinck, of the Université Libre de Bruxelles in Brussels, Belgium, and colleagues noted.

This paper reviews the available data on risdiplam, a novel drug designed to treat patients with SMA. This therapy consists of a survival motor neuron 2 premessenger RNA splicing modifier, which has been studied since 2014.

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Recent clinical trials reported a significant improvement regarding the symptoms and presentation of SMA during a 1-year follow-up period. Moreover, risdiplam achieved results comparable to other drugs, such as nusinersen and onasemnogene abeparvovec. Regardless, a comparative analysis is still pending.

On the other hand, risdiplam did not lead to drug-related adverse events for up to 4 years, with safety studies still ongoing.

“This up to now favorable efficacy/safety profile allows risdiplam to fulfill unmet needs in this recent era of new SMA therapies,” the authors highlighted.

One of the most important advantages of this drug is its oral administration and ability to cross the blood-brain barrier, the investigators noted. This characteristic allows patients to comply with therapy from home and does not require invasive procedures or hospitalization, further eliminating adverse events related to infusions, the authors noted.

The researchers also found a beneficial use of risdiplam in potentially all patients with SMA, and specifically those who have experienced side effects of current treatment or patients who do not comply with all criteria for treatment with onasemnogene abeparvovec.

However, research should continue to fill in some remaining knowledge gaps, the study authors noted. For example, longer-term results and safety profiles are yet to be determined. Finally, understanding the interactions and potentially synergistic effects of risdiplam with other drugs already available may lead to better therapeutic options for patients with SMA, they added.


Deconinck N, Devos E. Risdiplam as an orphan drug treatment of spinal muscular atrophy in adults and children (2 months or older). Expert Opin Orphan Drugs. Published online November 29, 2022. doi:10.1080/21678707.2022.2152671