How does risdiplam compare with other treatments for Types 1–3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison
Publication: Journal of Comparative Effectiveness Research
Abstract
Aim: To conduct indirect treatment comparisons between risdiplam and other approved treatments for spinal muscular atrophy (SMA). Patients & methods: Individual patient data from risdiplam trials were compared with aggregated data from published studies of nusinersen and onasemnogene abeparvovec, accounting for heterogeneity across studies. Results: In Type 1 SMA, studies of risdiplam and nusinersen included similar populations. Indirect comparison results found improved survival and motor function with risdiplam versus nusinersen. Comparison with onasemnogene abeparvovec in Type 1 SMA and with nusinersen in Types 2/3 SMA was challenging due to substantial differences in study populations; no concrete conclusions could be drawn from the indirect comparison analyses. Conclusion: Indirect comparisons support risdiplam as a superior alternative to nusinersen in Type 1 SMA.
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References
Papers of special note have been highlighted as: •• of considerable interest
1.
Verhaart IEC, Robertson A, Wilson IJ et al. Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy – a literature review. Orphanet J. Rare Dis. 12(1), 124 (2017).
2.
D'amico A, Mercuri E, Tiziano FD, Bertini E. Spinal muscular atrophy. Orphanet J. Rare Dis. 6, 71 (2011).
3.
Lefebvre S, Burglen L, Reboullet S et al. Identification and characterization of a spinal muscular atrophy-determining gene. Cell 80(1), 155–165 (1995).
4.
Burghes AH. When is a deletion not a deletion? When it is converted. Am. J. Hum. Genet. 61(1), 9–15 (1997).
5.
Lorson CL, Hahnen E, Androphy EJ, Wirth B. A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy. Proc. Natl Acad. Sci. USA 96(11), 6307–6311 (1999).
6.
Prior TW. Perspectives and diagnostic considerations in spinal muscular atrophy. Genet. Med. 12, 145–152 (2010).
7.
Singh RN, Howell MD, Ottesen EW, Singh NN. Diverse role of survival motor neuron protein. Biochim. Biophys. Acta Gene Regul. Mech. 1860(3), 299–315 (2017).
8.
Munsat TL, Davies KE. International SMA consortium meeting. (26–28 June 1992, Bonn, Germany). Neuromusc. Disord. 2(5–6), 423–428 (1992).
9.
Dubowitz V. Very severe spinal muscular atrophy (SMA type 0): an expanding clinical phenotype. Eur. J. Paediatr. Neurol. 3(2), 49–51 (1999).
10.
Finkel RS, Mcdermott MP, Kaufmann P et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. Neurology 83(9), 810–817 (2014).
11.
Mercuri E, Bertini E, Iannaccone ST. Childhood spinal muscular atrophy: controversies and challenges. Lancet Neurol. 11(5), 443–452 (2012).
12.
Kaufmann P, Mcdermott MP, Darras BT et al. Observational study of spinal muscular atrophy type 2 and 3: functional outcomes over 1 year. Arch. Neurol. 68(6), 779–786 (2011).
13.
Farrar MA, Vucic S, Johnston HM, Du Sart D, Kiernan MC. Pathophysiological insights derived by natural history and motor function of spinal muscular atrophy. J. Pediatrics 162(1), 155–159 (2013).
14.
Monani UR, De Vivo DC. Neurodegeneration in spinal muscular atrophy: from disease phenotype and animal models to therapeutic strategies and beyond. Future Neurol. 9(1), 49–65 (2014).
15.
Ratni H, Ebeling M, Baird J 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. 61(15), 6501–6517 (2018).
16.
EVRYSDI(R) Highlights of prescribing information. https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/213535s000lbl.pdf (September).
17.
Evrysdi (risdiplam) EMA prescribing information. https://www.ema.europa.eu/en/documents/product-information/evrysdi-epar-product-information_en.pdf (May).
18.
Baranello G, Darras BT, Day JW et al. Risdiplam in Type 1 spinal muscular atrophy. N. Engl. J. Med. 384(10), 915–923 (2021).
•• Reports the primary data from Part 1 of the FIREFISH trial, an open-label clinical trial of risdiplam in Type 1 SMA.
19.
Darras BT, Masson R, Mazurkiewicz-Bełdzińska M et al. Risdiplam-treated infants with Type 1 spinal muscular atrophy versus historical controls. N. Engl. J. Med. 385(5), 427–435 (2021).
•• Reports the primary data from an open-label clinical trial of risdiplam in early-onset (Type 1) SMA (FIREFISH Part 2).
20.
Mercuri E, Deconinck N, Mazzone E et al. Safety and efficacy of once-daily risdiplam in Type 2 and non-ambulant Type 3 spinal muscular atrophy: a Phase III, double-blind, randomised, placebo-controlled trial. Lancet Neurol., (2021).
•• Reports the primary data from a randomized, double-blind, placebo-controlled clinical trial of risdiplam in Type 2 and non-ambulant Type 3 SMA (SUNFISH Part 2).
21.
SPINRAZA® (nusinersen) EMA prescribing information. http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Product_Information/human/004312/WC500229704.pdf (November).
22.
SPINRAZA® (nusinersen) US prescribing information. https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/209531lbl.pdf (November).
23.
NCT02193074: a study to assess the efficacy and safety of ISIS-SMNRx in infants with spinal muscular atrophy (ENDEAR). https://clinicaltrials.gov/ct2/show/NCT02193074 (July).
24.
NCT02292537: a study to assess the efficacy and safety of nusinersen (ISIS 396443) in participants with later-onset spinal muscular atrophy (SMA) (CHERISH). https://clinicaltrials.gov/ct2/show/NCT02292537 (Accessed December 2020).
25.
ZOLGENSMA® (onasemnogene abeparvovec-xioi) US prescribing information. https://www.fda.gov/media/126109/download (November).
26.
ZOLGENSMA® (onasemnogene abeparvovec-xioi). https://www.ema.europa.eu/en/medicines/human/EPAR/zolgensma (November).
27.
Mendell J. Single-dose gene-replacement therapy for spinal muscular atrophy. N. Engl. J. Med. 377, 1713–1722 (2017).
28.
Mendell JR, Al-Zaidy SA, Lehman KJ et al. Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy. JAMA Neurol. 78(7), 834–841 (2021).
29.
Day JW, Finkel RS, Chiriboga CA et al. Onasemnogene abeparvovec gene therapy for symptomatic infantile-onset spinal muscular atrophy in patients with two copies of SMN2 (STR1VE): an open-label, single-arm, multicentre, Phase III trial. Lancet Neurol. 20(4), 284–293 (2021).
•• This study reports the primary data from an open-label clinical trial of onasemnogene abeparvovec in early-onset (Type 1) SMA (STR1VE-US).
30.
Finkel RS, Dabbous O, Arjunji R et al. P.060 Time to treatment effect in spinal muscular atrophy Type 1 (SMA1): an indirect comparison of treatments. Can. J. Neurologic. Sci. 46, S30 (2019).
31.
Dabbous O, Maru B, Jansen JP et al. Survival, motor function, and motor milestones: comparison of AVXS-101 relative to nusinersen for the treatment of infants with spinal muscular atrophy Type 1. Adv. Ther. 36(5), 1164–1176 (2019).
32.
Liao S, Bohn J, De Moor C, Xaplanteris X, Farwell W, Padaris A. PR109 a cautionary tale for indirect treatment comparisons: an example from infantile-onset spinal muscular atrophy. Value Health 23, (2020).
33.
Bischof M, Lorenzi M, Lee J, Druyts E, Balijepalli C, Dabbous O. Matching-adjusted indirect treatment comparison of onasemnogene abeparvovec and nusinersen for the treatment of symptomatic patients with spinal muscular atrophy Type 1. Curr. Med. Res. Opin. (2021) (Epub ahead of print).
34.
Bucher HC, Guyatt GH, Griffith LE, Walter SD. The results of direct and indirect treatment comparisons in meta-analysis of randomized controlled trials. J. Clin. Epidemiol. 50(6), 683–691 (1997).
35.
Phillippo DM, Ades AE, Dias S, Palmer S, Abrams KR, Welton NJ. NICE DSU technical document 18: methods for population-adjusted indirect comparisons in submissions to NICE. (2016).
36.
Baranello G, Gorni K, Daigl M et al. Prognostic factors and treatment-effect modifiers in spinal muscular atrophy. Clin. Pharmacol. Therapeut., (2021) (Epub ahead of print).
•• Reports on the prognostic and predictive factors and treatment-effect modifiers for Types 1–3 SMA through a systematic literature review.
37.
Glanzman AM, Mazzone E, Main M et al. The Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND): test development and reliability. Neuromusc. Disord. 20, 155–161 (2010).
38.
Pechmann A, Langer T, Schorling D et al. Evaluation of children with SMA Type 1 under treatment with nusinersen within the expanded access program in Germany. J. Neuromuscul. Dis. 5(2), 135–143 (2018).
39.
Annoussamy M, Seferian A, Daron A et al. Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study. Ann. Clin. Transl. Neurol. 8(2), 359–373 (2021).
40.
Mercuri E, Lucibello S, Perulli M et al. Longitudinal natural history of Type I spinal muscular atrophy: a critical review. Orphanet J. Rare Dis. 15(1), 84 (2020).
41.
Haaker G, Fujak A. Proximal spinal muscular atrophy: current orthopedic perspective. App. Clin. Genet. 6(11), 113–120 (2013).
42.
NCT02913482: a study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in infants with Type 1 spinal muscular atrophy (FIREFISH). https://clinicaltrials.gov/ct2/show/NCT02913482 (July).
43.
NCT03306277: gene replacement therapy clinical trial for patients with spinal muscular atrophy Type 1 (STR1VE-US). https://clinicaltrials.gov/ct2/show/NCT03306277?term=AVXS-101&rank=2 (July).
44.
NCT01839656: a study to assess the safety and pharmacokinetics of ISIS SMNRx in infants with spinal muscular atrophy. https://clinicaltrials.gov/ct2/show/NCT01839656
45.
NCT02122952: gene transfer clinical trial for spinal muscular atrophy Type 1. https://clinicaltrials.gov/ct2/show/NCT02122952 (July).
46.
NCT03837184: single-dose gene replacement therapy using for patients with spinal muscular atrophy Type 1 with one or two SMN2 copies (STR1VE-AP). https://clinicaltrials.gov/ct2/show/NCT03837184?term=NCT03837184&draw=2&rank=1 (Accessed 2020).
47.
NCT03461289: single-dose gene replacement therapy clinical trial for patients with spinal muscular atrophy Type 1 (STRIVE-EU). https://clinicaltrials.gov/ct2/show/NCT03461289?term=NCT03461289&draw=2&rank=1 (Accessed 2020).
48.
NCT02268552: an open-label study of LMI070 in Type 1 spinal muscular atrophy (SMA). https://clinicaltrials.gov/ct2/show/NCT02268552
49.
NCT02908685: a study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics and efficacy of RO7034067 in Type 2 and 3 spinal muscular atrophy participants (SUNFISH). https://clinicaltrials.gov/ct2/show/NCT02908685 (July).
50.
Darras B, Chiriboga C, Swoboda K et al. Results of a Phase 2 study of ISIS-SMNRx in children with spinal muscular atrophy. Neuromusc. Disord. 24(9–10), 920 (2014).
51.
Chiriboga CA, Swoboda KJ, Darras BT et al. Results from a Phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy. Neurology 86(10), 890–897 (2016).
52.
NCT01703988: an open-label safety, tolerability and dose-range finding study of multiple doses of ISIS SMNRx in patient with spinal muscular atrophy (SMNRx - CS2). https://clinicaltrials.gov/ct2/show/NCT01703988
53.
NCT02052791: an open-label safety and tolerability study of nusinersen (ISIS 396443) in participants with spinal muscular atrophy (SMA) who previously participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (Accessed August 2020).
54.
NCT01302600: safety and efficacy of olesoxime (TRO19622) in 3–25 years SMA patients. https://clinicaltrials.gov/ct2/show/NCT01302600 (Accessed June 2020).
55.
NCT03032172: a study of RO7034067 in adult and pediatric participants with spinal muscular atrophy (JEWELFISH). https://clinicaltrials.gov/ct2/show/NCT03032172 (July).
56.
NCT03921528: an active treatment study of SRK-015 in patients with Type 2 or Type 3 spinal muscular atrophy (TOPAZ). https://clinicaltrials.gov/ct2/show/NCT03921528 (Accessed September 2020).
57.
NCT04089566: study of nusinersen (BIIB058) in participants with spinal muscular atrophy (DEVOTE). https://www.clinicaltrials.gov/ct2/show/NCT04089566
58.
NCT02462759: a study to assess the safety and tolerability of nusinersen (ISIS 396443) in participants with spinal muscular atrophy (SMA). (EMBRACE). https://www.clinicaltrials.gov/ct2/show/NCT02462759 (Accessed July 2021).
59.
NCT02594124: a study for participants with spinal muscular atrophy (SMA) who previously participated in nusinersen (ISIS 396443) investigational studies. (SHINE). https://clinicaltrials.gov/ct2/show/NCT02594124 (July).
60.
NCT04042025: long-term follow-up study of patients receiving onasemnogene abeparvovec-xioi. https://www.clinicaltrials.gov/ct2/show/NCT04042025 (Accessed July 2021).
61.
Rudnicki S, Andrews J, Malik F, Wolff A, Day. CK-2127107 a selective activator of the fast skeletal muscle troponin complex, for the potential treatment of spinal muscular atrophy. Amyotroph. Lateral Scler. Frontotemporal Degener. 17(1), 241 (2016).
62.
NCT02628743: a study to evaluate long term safety, tolerability, and effectiveness of olesoxime in patients with spinal muscular atrophy. https://clinicaltrials.gov/ct2/show/NCT02628743 (November).
63.
NCT03381729: study of intrathecal administration of AVXS-101 for spinal muscular atrophy (STRONG). https://clinicaltrials.gov/ct2/show/NCT03381729?term=strong&cond=Spinal+Muscular+Atrophy&rank=1
64.
Day J, Chiriboga C, Crawford T et al. P.349Onasemnogene abeparvovec gene-replacement therapy (GRT) for spinal muscular atrophy Type 1 (SMA1): pivotal Phase III study (STR1VE) update. Neuromusc. Disord. 29, S183 (2019).
65.
Finkel RS, Mercuri E, Darras BT et al. Nusinersen versus sham control in infantile-onset spinal muscular atrophy. N. Engl. J. Med. 377(18), 1723–1732 (2017).
•• Reports the primary data from a randomized, sham-controlled clinical trial of nusinersen in early-onset (Type 1) SMA (ENDEAR).
66.
European Medicines Agency CFMPFHU, Committee for Advanced Therapies. Assessment report: zolgensma (2020). https://www.ema.europa.eu/en/documents/assessment-report/zolgensma-epar-public-assessment-report_en.pdf
67.
Mercuri E, Darras BT, Chiriboga CA et al. Nusinersen versus sham control in later-onset spinal muscular atrophy. N. Engl. J. Med. 378, 625–635 (2018).
•• Reports the primary data from a randomized, sham-controlled clinical trial of nusinersen in later-onset (Types 2 and 3) SMA (CHERISH).
68.
Signorovitch JE, Wu EQ, Yu AP et al. Comparative effectiveness without head-to-head trials: a method for matching-adjusted indirect comparisons applied to psoriasis treatment with adalimumab or etanercept. Pharmacoeconomics 28(10), 935–945 (2010).
69.
Phillippo DM, Ades AE, Dias S, Palmer S, Abrams KR, Welton NJ. Methods for population-adjusted indirect comparisons in health technology appraisal. Med. Decis. Making 38(2), 200–211 (2018).
70.
Signorovitch JE, Sikirica V, Erder MH et al. Matching-adjusted indirect comparisons: a new tool for timely comparative effectiveness research. Value Health 15(6), 940–947 (2012).
71.
Daigl M, Kotzeva A, Gorni K et al. PRO12: how does risdiplam compare in infantile-onset spinal muscular atrophy (SMA)? Preliminary indirect treatment comparisons based on FIREFISH Part 1 data. Value Health 22, S843 (2019).
72.
Phillippo DM, Dias S, Ades AE et al. Multilevel network meta-regression for population-adjusted treatment comparisons. J. R. Stat. Soc. Ser. A Stat. Soc. 183(3), 1189–1210 (2020).
73.
Wood L, Egger M, Gluud LL et al. Empirical evidence of bias in treatment effect estimates in controlled trials with different interventions and outcomes: meta-epidemiological study. BMJ 336(7644), 601–605 (2008).
74.
Mercuri E, Finkel RS, Muntoni F et al. Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care. Neuromusc. Disord. 28(2), 103–115 (2018).
75.
Finkel RS, Mercuri E, Meyer OH et al. Diagnosis and management of spinal muscular atrophy: Part 2: Pulmonary and acute care; medications, supplements and immunizations; other organ systems; and ethics. Neuromusc. Disord. 28(3), 197–207 (2018).
76.
Mercuri E, Lucibello S, Pera MC et al. Long-term progression in type II spinal muscular atrophy: a retrospective observational study. Neurology 93(13), e1241–e1247 (2019).
77.
Merlini L, Bertini E, Minetti C et al. Motor function-muscle strength relationship in spinal muscular atrophy. Muscle Nerve 29(4), 548–552 (2004).
78.
Millino C, Fanin M, Vettori A et al. Different atrophy-hypertrophy transcription pathways in muscles affected by severe and mild spinal muscular atrophy. BMC Med. 7, 14 (2009).
79.
Swallow E, Fang A, Signorovitch JE, Plumb J, Borghs S. Can matching-adjusted indirect comparison methods mitigate placebo response differences among patient populations in adjunctive trials of brivaracetam and levetiracetam? CNS Drugs 31(10), 899–910 (2017).
80.
Cooper M, Kilvert H, Hodgkins P, Roskell N, Eldar-Lissai A. Using matching-adjusted indirect comparisons and network meta-analyses to compare efficacy of brexanolone injection with selective serotonin reuptake inhibitors for treating postpartum depression. CNS Drugs 33(10), 1039–1052 (2019).
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How does risdiplam compare with other treatments for Types 1–3 spinal muscular atrophy: a systematic literature review and indirect treatment comparison. (2022) Journal of Comparative Effectiveness Research. DOI: 10.2217/cer-2021-0216
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