New Zolgensma data demonstrate age-appropriate development when used presymptomatically and rapid, clinically meaningful efficacy in symptomatic children, even those with severe SMA at baseli

- One hundred percent of patients (14/14) met the secondary endpoint of survival without ventilatory support of any kind at 14 months of age,4?versus only 26 percent of patients in the Pediatric Neuromuscular Clinical Research (PNCR) natural history cohort.6
- All patients (100 percent) achieved the primary endpoint of sitting independently for at least 30 seconds, including 11 (79 percent) who achieved this milestone within the WHO window of normal development.4
- Eleven patients (79 percent) could stand independently, seven of whom achieved this milestone within the WHO window of normal development.4
- Nine patients (64 percent) could walk independently, five of whom achieved this milestone within the WHO window of normal development.4
- All patients (100 percent) were independent of nutritional and respiratory support for the duration of the study.4
- Nearly all patients (13/14) achieved the additional secondary efficacy endpoint of age-appropriate weight maintenance without non-oral feeding support at any visit up to 18 months of age.4
- All patients (100 percent) achieved or maintained a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) score of =58.4?According to natural history, untreated patients with SMA Type 1 almost never achieve a CHOP INTEND score of =40.6
- All patients (100 percent) had Bayley-III fine motor performance scores similar to same-age peers without SMA and the majority (64 percent) had gross motor performance scores similar to same-age peers without SMA.4
- Thirty-three patients were enrolled; and 32 patients completed the study.
- Twenty-seven of 33 patients (82 percent) achieved developmental motor milestones not observed in the natural history of SMA Type 1.5
- Fourteen of 32 patients (44 percent) in the intention-to-treat (ITT) population achieved the primary endpoint of sitting independently for =10 seconds, observed at a median age of 15.9 months (7.7?18.6). The patient who was not in the ITT population also achieved this primary outcome measure.5
- Twenty-three of 30 patients (77 percent) achieved head control (three already had control at baseline), 19 of 33 (58 percent) rolled from back to sides and 16 patients (49 percent) sat without support for =30 seconds, including all patients who met the primary endpoint of sitting without support for =10 seconds.5
- In addition, two patients stood with assistance, and one crawled, stood, pulled to stand, and walked without assistance, all by 18 months of age.5
- Thirty-one of 32 patients (97 percent) achieved the secondary efficacy endpoint of survival, free from permanent ventilatory support at 14 months,5?compared with a quarter of patients (26 percent) in the PNCR natural history cohort.6
- The majority of patients (73 percent) achieved a CHOP INTEND score of =40 points, 14 (42 percent) achieved a score of =50 points and three (9 percent) achieved a score of =58 points.5?According to natural history, untreated patients with SMA Type 1 almost never achieve a CHOP INTEND score =40.6
- Thirteen of 33 patients (39 percent) remained independent of any type of daily ventilatory support at 18 months of age,5?compared with none in the PNCR data set.6
- Of the nine patients who required ventilatory support at baseline, two achieved independence, and of the 24 patients who did not require ventilatory support at baseline, a majority of patients (67 percent) remained free from this support at the end of the study.5
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References- Novartis. 2021. Q1 2021 Results Investor presentation [PowerPoint Presentation]. Available at:?https://www.novartis.com/sites/www.novartis.com/files/q1-2021-investor-presentation.pdf. Accessed May 2021
- Anderton RS and Mastaglia FL.?Expert Rev Neurother. 2015;15(8):895?908.
- Finkel RS, McDermott MP, Kaufmann P. et al. Observational study of spinal muscular atrophy type I and implications for clinical trials.?Neurology. 2014;83(9):810-7.
- Strauss K. et al. Onasemnogene Abeparvovec for Presymptomatic Infants with Spinal Muscular Atrophy and 2 Copies of SMN2: A Phase III Study. Abstract presented at the 7th?Congress of the European Academy of Neurology. 19?22 June 2021.
- Mercuri E. et al. Gene replacement therapy for symptomatic spinal muscular atrophy type 1: final results of the Phase III STR1VE-EU study. Abstract presented at the 7th?Congress of the European Academy of Neurology. 19?22 June 2021.
- Pediatric Neuromuscular Clinical Research. PNCR Database: Weights (kg) by Patient Ages (Months) for SMA Types I, II, and III All Visits.
- National Organization for Rare Disorders (NORD). Spinal Muscular Atrophy. Available at:?http://rarediseases.org/rarediseases/spinal-muscular-atrophy/. Accessed May 2021.
- Soler-Botija C, et al. Brain. 2002;125(7):1624-1634.
- Wang CH, et al. J Child Neurol. 2007;22(8):1027-1049.
- Darras BT, Finkel RS. ?Natural history of spinal muscular atrophy.? In: Sumner CJ, Paushkin S, Ko CP, eds. Spinal Muscular Atrophy: Disease Mechanisms and Therapy, 2nd ed. London, UK: Academic Press/Elsevier;2017:399-421.
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