Biogen Plans to Initiate Phase 3b Study Evaluating Potential Benefit of a Higher Dose of Nusinersen in Patients Previously Treated with Evrysdi? (risdiplam)
- Clinical data available to date suggest there are remaining unmet needs in some spinal muscular atrophy (SMA) patients treated with Evrysdi1-3
- The ASCEND study aims to evaluate whether treatment with a higher dose of nusinersen has the potential to improve clinical outcomes in these patients
- Building on the proven efficacy and well-established safety of the currently approved 12-milligram dose, patients in this study will receive an investigational higher dose of nusinersen, which is also being evaluated in the DEVOTE study
- Mercuri E. et al. SUNFISH Part 2: Efficacy and safety of risdiplam (RG7916) in patients with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA). SMA Europe Feb 5-7, 2020 presentation.
- Evrysdi US FDA Summary Basis of Approval (SBA) documents. Clinical Review(s): Page 67, table 15. Available at:?https://www.accessdata.fda.gov/drugsatfda_docs/nda/2020/213535Orig1s000TOC.cfm. Accessed: September 2021.
- European Medicines Agency: EMA/216061/2021 ? Evrysdi EPAR. Page 195, table 41. Available at:?https://www.ema.europa.eu/en/documents/assessment-report/evrysdi-epar-public-assessment-report_en.pdf. Accessed September 2021.
- Rouault F, et al. Disease impact on general well-being and therapeutic expectations of European Type II and Type III spinal muscular atrophy patients. Neuromuscul Disord. 2017 May;27(5):428-438. doi: 10.1016/j.nmd.2017.01.018. Epub 2017 Feb 3.
- Mazzone ES, et al. Revised upper limb module for spinal muscular atrophy: Development of a new module. Muscle Nerve. 2017 Jun;55(6):869-874. doi: 10.1002/mus.25430. Epub 2017 Feb 6.
- Evrysdi US FDA SBA documents. Clinical Pharmacology Review(s): Page 41, table 18. The approximately 40% reduction is the percentage difference between risdiplam observed exposures in the youngest infants and adults. Available at:?https://www.accessdata.fda.gov/drugsatfda_docs/nda/2020/213535Orig1s000TOC.cfm. Accessed: September 2021.
- European Medicines Agency: EMA/216061/2021 ? Evrysdi EPAR. Page 71, figure 13. The approximately 40% reduction is the percentage difference between risdiplam observed exposures in the youngest infants and adults. Available at:?https://www.ema.europa.eu/en/documents/assessment-report/evrysdi-epar-public-assessment-report_en.pdf. Accessed September 2021.
- Evrysdi U.S. Prescribing Information. Available at:?https://www.gene.com/download/pdf/evrysdi_prescribing.pdf. Accessed: September 2021.
- Finkel RS, et al. Scientific Rationale for a Higher Dose of Nusinersen. Cure SMA Virtual Research and Clinical Care Meeting June 9?11, 2021 poster presentation.
- Core Data Sheet, Version 9, January 2019. SPINRAZA. Biogen Inc, Cambridge, MA.
- National Institute of Neurological Disorders and Stroke, NIH. Spinal Muscular Atrophy Fact Sheet. Available at?https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Spinal-Muscular-Atrophy-Fact-Sheet. Accessed: September 2021.
- Wadman RI, Wijngaarde CA, Stam M, et al. Muscle strength and motor function throughout life in a cross-sectional cohort of 180 patients with spinal muscular atrophy types 1c?4. Eur J Neurol. 2018;25(3):512-518.
- Cure SMA. About SMA. Available at?https://www.curesma.org/about-sma/. Accessed: September 2021.
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