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    Risdiplam

    Other names: RG7916; RO7034067

    Modality: SMN2 Pre-mRNA Splicing Modifier

    Disease State: Spinal Muscular Atrophy (SMA)

    Summary

    Risdiplam is an investigational survival motor neuron 2 (SMN2) splicing modifier indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.1 Ongoing clinical studies will further evaluate long-term benefits, real-world effectiveness, and optimal place in therapy alongside existing treatment options.

    Clinical Trials

    Risdiplam
    NCT ID Study Phase Enrollment Status Study Title
    NCT05861986 Phase IV Recruiting A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy View details
    NCT05861999 Phase IV Recruiting A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy View details
    NCT03779334 Phase II Active, not recruiting A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy View details
    NCT05232929 Phase IV Active, not recruiting Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) View details

    Proposed Mechanism of Action

    Selective SMN2 splicing modifier designed to bind with specificity to SMN2 pre-mRNA and promote inclusion of exon 7, thus increasing the production of functional SMN protein. It penetrates the blood-brain barrier providing  systemic distribution.2-4

    Risdiplam is proposed to have a high specificity for SMN2 pre-mRNA2,4

    • The proposed mechanism of action for risdiplam is based on studies with small molecules closely related to risdiplam2,4
    • Risdiplam is proposed to bind to 2 sites on SMN2 pre-mRNA2:
      • 5' splice site (5'ss) of intron 7
      • Exonic splicing enhancer 2 (ESE2) in exon 7 
    • Binding to the 5’ss of intron 7 improves recognition by the U1 snRNP, promoting inclusion of exon 72
    • The unique specificity of 2 pre-mRNA binding sites increases levels of full-length SMN mRNA and protein while reducing the impact on splicing of other pre-mRNA2-4
    1. Roche. Product development pipeline. Roche. January 29, 2026. Accessed February 8, 2026. https://www.roche.com/​solutions/​pipeline
    2. Sivaramakrishnan M, McCarthy KD, Campagne S, et al. Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers. Nat Commun. 2017;8(1):1476. doi:10.1038/​s41467-017-01559-4
    3. Baranello G, Darras BT, Day JW, et al. Risdiplam in type 1 spinal muscular atrophy. N Engl J Med. 2021;384(10):915-923. doi:10.1056/​NEJMoa2009965
    4. Mercuri E, Baranello G, Boespflug-Tanguy O, et al. Risdiplam in types 2 and 3 spinal muscular atrophy: A randomised, placebo-controlled, dose-finding trial followed by 24 months of treatment. Eur J Neurol. 2023;30(7):1945-1956. doi:10.1111/​ene.15499
    5. Swoboda KJ. Of SMN in mice and men: a therapeutic opportunity. J Clin Invest. 2011;121(8):2978-2981. doi:10.1172/​JCI58752

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    TOP

    • 5'ss
      5' splice site

    • ESE2
      Exonic splicing enhancer 2

    • SMA
      Spinal muscular atrophy

    • SMN
      Survival motor neuron

    • SMN2
      Survival motor neuron 2

    • mRNA
      Messenger RNA

    • snRNP
      Small nuclear ribonucleoproteins

    +