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A Second Look: Discussing the Spinal Muscular Atrophy Space- with Zolgensma and Spinraza in the spotlightTicker(s): NVS, BIIB
Name: Dr Edward Smith - MD
Institution: Duke University
- Co-Director of Duke Children's Neuromuscular Program & Professor of Neurology and Pediatrics at Duke.
- Treats hundreds of neuromuscular patients, including over 60 Spinal Muscular Atrophy patients and 80 Duchenne MD patients (all ages).
- Has administered over 250+ doses of Spinraza since April 2017.
- Actively involved in clinical research and trials with a focus on neuromuscular diseases; PI on multiple DMD studies including an Exon 53 skipping specific study.
Please tell us about your clinical practice, background in treating Spinal Muscular Atrophy, and research in the space. How many patients do you treat,what is the first line of therapy? What % of patients do not respond well to first line?
How often have you been prescribing Zolgensma for children under 2 since its approval last May? How does it compare as a one-dose treatment vs other options you might have?
- How do you comment on the recent FDA partial hold on Zolgensma intrathecal clinical trials for SMA following animal findings showing dorsal root ganglia mononuclear cell inflammation, accompanied by neuronal cell body degeneration or loss?
- Also, a few months ago, the FDA had also addressed some data integrity issues with the BLA of Zolgensma. Reasons for potential trouble down the road?
Added By: c_admin
- Data from START study of Zolgensma had 7/15 children in the high-dose group becoming completely independent of ventilatory assistance. 11 had achieved and retained the ability to swallow independently, and four were able to feed orally. And 9 were able to sit unaided for at least 30 seconds. How does this datapoint compare to Spinraza?
Added By: c_admin
40% of 82 patients treated with Spinraza achieved improvement in motor milestones in the interim analysis prior to its FDA Approval in 2016. How likely is Zolgensma to gain approval for adults as well, considering this?Added By: c_admin
How does Zolgensma and Spinraza’s mechanism of action differ from one another?
13 of the 18 patients (72.2%) enrolled in Phase 3 SPR1NT experienced at least one treatment-emergent adverse event and 7 (38.9%) were reported to have a TEAE considered by the investigator to be related to Zolgensma. How does this AE outcome compare to Spinraza’s?Added By: c_admin
How likely would you be to switch adult patients to Zolgensma in the eventuality of an FDA approval in the future?
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