AveXis data reinforce effectiveness of Zolgensma in treating spinal muscular atrophy (SMA) Type 1
- Ph 3 STR1VE data show prolonged event-free survival, early and rapid increases in CHOP-INTEND and significant milestone achievement in SMA Type 1, consistent with START trial
- First-in-human biodistribution data?show transduction in intended CNS targets and widespread SMN expression comparable to tissue from unaffected control?
- More than 150 patients treated with Zolgensma, only 5% of screened patients up to 5 years old excluded due to AAV9 antibody titers greater than 1:50
?Milestone Achieved, n (%)a | Phase 3 STR1VE Study, n=22 | |
September 27, 2018 | December 31, 2018 | |
Holds head erect >=3 seconds without supportb | 12 (54.5)c | 17 (77.3) |
Turns from back to both right and left sided | 3 (13.6) | 7 (31.8) |
Sits without support for >=30 secondse | 3 (13.6) | 8 (36.4) |
Stands with assistancef | 0 | 1 (4.5) |
Median duration of follow-up at data cut | 5.5 months | 8.1 months |
Median age at data cut | 9.4 months | 12.5 months |
Patients older than 12 months, n (%) | 5 (22.7) | 13 (59.1) |
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Novartis Media Relations Central media line: +41 61 324 2200 E-mail:?media.relations@novartis.comEric Althoff Novartis Global Media Relations +41 61 324 7999 (direct) +41 79 593 4202 (mobile) eric.althoff@novartis.com | Farah Bulsara Speer VP, Corporate Communications, AveXis +1 312 543 2881 (mobile) fSpeer259@avexis.com |