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Review

Risdiplam

No authors listed
In: LiverTox: Clinical and Research Information on Drug-Induced Liver Injury [Internet]. Bethesda (MD): National Institute of Diabetes and Digestive and Kidney Diseases; 2012.
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Review

Risdiplam

No authors listed.
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No abstract available

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References

    1. FDA. 2020. Clinical Review. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2020/213535Orig1s000M...(FDA website with product label and clinical review of the data on efficacy and safety of risdiplam in support of its approval as therapy of spinal muscular atrophy mentions that “there were no clinically significant differences in key blood chemistry parameters between the placebo and risdiplam groups” including ALT, AST, and bilirubin values and no instance of clinically apparent liver injury with jaundice).
    1. Kolb SJ, Coffey CS, Yankey JW, Krosschell K, Arnold WD, Rutkove SB, Swoboda KJ, et al.; NeuroNEXT Clinical Trial Network on behalf of the NN101 SMA Biomarker Investigators. Natural history of infantile-onset spinal muscular atrophy. Ann Neurol 2017; 82: 883-91.(Natural history study of 26 infants with SMA1 demonstrating the rate of motor function deterioration, respiratory failure, and survival in untreated infants). - PMC - PubMed
    1. Ratni H, Ebeling M, Baird J, Bendels S, Bylund J, Chen KS, Denk N, et al. Discovery of risdiplam, a selective survival of motor neuron-2 (SMN2) gene splicing modifier for the treatment of spinal muscular atrophy (SMA). J Med Chem. 2018;61:6501-6517.(Description of the development of risdiplam, the optimization of its efficacy in splice modification of SMN2 with minimalization of its modification of mRNAs of other important proteins [FOXM1] and improvement in pharmacokinetics and safety in preclinical studies). - PubMed
    1. Stevens D, Claborn MK, Gildon BL, Kessler TL, Walker C. Onasemnogene abeparvovec-xioi: gene therapy for spinal muscular atrophy. Ann Pharmacother 2020; 54: 1001-9.(Review of the mechanism of action, clinical efficacy and safety of onasemnogene abeparvovec mentions that two treatment related serious adverse events occurred in the initial trial, elevations in serum ALT levels of 14 and 31 times upper limit of normal that resolved with corticosteroid therapy; no further hepatic adverse events being reported in clinical trials and registration studies, although the product has a black box warning about liver injury and prophylactic corticosteroids are recommended). - PubMed
    1. Dhillon S. Risdiplam: first approval. Drugs. 2020;80:1853-1858.(Summary of the chemical structure, mechanism of action, history of development, pharmacology, clinical efficacy, and safety of risdiplam shortly after its approval in the US, does not mention ALT elevations or hepatotoxicity). - PubMed

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