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Risdiplam (Evrysdi°) in spinal muscular atrophy. Continue the evaluation

FEATURED REVIEW Risdiplam has been authorised in the European Union for use in patients aged 2 months or older with spinal muscular atrophy. How do Prescrire's editors rate this drug in this situation?
Full review (3 pages) available for download by subscribers.

Prescrire's rating

  •  JUDGEMENT RESERVED  In infants with symptomatic type 1 spinal muscular atrophy, risdiplam appears to prolong survival, reduce respiratory support requirements and improve certain motor functions, when compared with "historical controls". However, marked disability remained, and the drug’s effect beyond 3 years is unknown. Risdiplam is of dubious efficacy in types 2 and 3 spinal muscular atrophy. Its main adverse effects in trials were gastrointestinal disorders, skin disorders and infections, some of which were serious. There are many uncertainties concerning its long-term efficacy and harms. As risdiplam is taken orally, it may appear more straightforward to administer than other drugs for spinal muscular atrophy. The evidence available in early 2022 appears to justify continued evaluation of this treatment in clinical trials. 

©Prescrire 1 Novembre 2022

Source: "Risdiplam (Evrysdi°) in spinal muscular atrophy. Continue the evaluation" Prescrire International 2022; 31 (242): 257-259. Subscribers only.

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See also:

"Onasemnogene
abeparvovec (Zolgensma°)
in spinal muscular atrophy.
Results that encourage
continued evaluation"
Prescrire Int 2021;
30 (231): 257-258.
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"Nusinersen in spinal
muscular atrophy.
Insufficient data"
Prescrire Int 2018;
27 (199): 290.
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"Zolgensma°: the drug
of extremes"
(April 2020)
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