Nusinersen Shows Long-Term SMA Success, Positive Data for an Investigational DMD Therapy, Zogenix to Resubmit Fenfluramine NDA


Neurology News Network for the week ending July 6, 2019.

This week, Neurology News Network covered a new analysis of NURTURE study data on nusinersen (Spinraza) in presymptomatic patients with spinal muscular atrophy (SMA), successful phase 1b findings for Pfizer's PF-06939926, an investigational gene therapy designed to treat Duchenne muscular dystrophy (DMD), and Zogenix's announcement that it intends to resubmit an NDA for fenfluramine (Finlepta) for Dravet syndrome following the receipt of the final minutes from the FDA following a Type A meeting. (transcript below).


Welcome to Neurology News Network. I’m Matt Hoffman. Let’s get into the news from this week.

Results from additional analysis of the NURTURE study reveal that treatment with nusinersen—marketed as Spinraza by Biogen—shows prolonged efficacy and safety in presymptomatic pediatric patients with spinal muscular atrophy after 45 months.

Presented at the Cure SMA Annual SMA Conference and the 5th Congress of the European Academy of Neurology, the findings ultimately showed that 100% of the 25 infants treated with nusinersen were able to sit without support while 88% were able to walk independently. Notably, these are milestones which are not documented in the natural history of SMA.

Recently, Pfizer presented data from a phase 1b clinical trial of PF-06939926, an investigational gene therapy designed to treat Duchenne muscular dystrophy at the 25th Annual Parent Project Muscular Dystrophy Connect Conference. The data, which are preliminary, show that at 2 months post-dosing, there are detectable mini-dystrophin immunofluorescence signals with the therapy. An average of 38% positive fibers were collected from those who were administered the study drug at 1E14 vg/kg and a mean of 69% positive fibers were taken from participants who received it at 3E14 vg/kg.

Zogenix announced that it has received the final minutes from the FDA following a Type A meeting on its investigational treatment for Dravet syndrome, fenfluramine, marketed as Fintepla, and has reported that it intends to resubmit its new drug application to the agency in the third quarter of 2019.

The meeting followed a refusal to file letter which the FDA sent to Zogenix regarding fenfluramine (also known as ZX008) in which 2 issues were identified—the lack of non-clinical data on the chronic administration of the treatment and an incorrect dataset included in the application. Zogenix president and CEO Stephen J. Farr, PhD, expressed his satisfaction with the meeting to NeurologyLive®.

For more direct access to expert insight, head to This has been Neurology News Network. Thanks for watching.

Related Videos
 Jocelyn Y. Cheng, MD
Opeolu Adeoye, MD, MS; Andrew Barerrto, MD, MS
David Z. Rose, MD
Shadi Yaghi, MD, associate professor of neurology at Brown University
© 2024 MJH Life Sciences

All rights reserved.