Novartis? Zolgensma (onasemnogene abeparvovec) Receives MHLW?s Approval for Spinal Muscular Atrophy
[caption id="attachment_9277" align="aligncenter" width="747"] Press Release[/caption]
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- Zolgensma??(onasemnogene abeparvovec), a one-time administered gene therapy, is approved for the treatment of SMA in patients under the age of two, including those who are pre-symptomatic at diagnosis
- Zolgensma has demonstrated significant and clinically meaningful therapeutic benefit in symptomatic and pre-symptomatic SMA, including prolonged event-free survival and achievement of motor milestones unseen in natural history of the disease?
- Designated as an intractable disease in Japan, SMA is the leading genetic cause of infant death and, if left untreated in its most common form (Type 1), leads to death or the need for permanent ventilation by the age of two in more than 90% of cases1,2
- Reimbursement with MHLW is expected by the end of 1H20, pending agreement, Zolgensma will be available at that time
- Farrar MA, et al.?Ann Neurol. 2017;81(3):355-368.
- 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
- Anderton RS and Mastaglia FL.?Expert Rev Neurother. 2015;15(8):895-908.
- Data on file.
- Verhaart IEC, Robertson A, et al. A multi-source approach to determine SMA incidence and research ready population.?J Neurol. 2017 Jul;264(7):1465-1473Soler-Botija C, et al.?Brain. 2002;125(7):1624-1634.
- 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 Spinal Muscular Atrophy. Chapter 25 - Natural History of Spinal Muscular Atrophy. October 2017.
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Novartis Media Relations E-mail:?media.relations@novartis.comAnja von Treskow Novartis External Communications +41 79 392 8697 (mobile) anja.von_treskow@novartis.com Eric Althoff Novartis US External Communications +1 646 438 4335 (mobile) eric.althoff@novartis.com | Farah Bulsara Speer SVP, Corporate Communications, AveXis +1 312 543 2881 (mobile) fSpeer259@avexis.com Seiko Adachi Country Comms Head, Head of Comms Pharma +81 36 899 5539 (direct) seiko.adachi@novartis.com |
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