FDA Approves Zolgensma for Spinal Muscular Atrophy, Nerivio Migra Device Granted De Novo Clearance for Migraine, Cladribine Reduces MRI Lesions Across Subgroups in ORACLE-MS Study
Neurology News Network for the week ending June 1, 2019.
PUBLISHED June 01, 2019
This week, Neurology News Network covered the FDA approval of Zolgensma (AveXis) for treatment of spinal muscular atrophy in pediatric patients aged 2 and younger, the FDA De Novo clearance to Theranica's Nerivio Migra for acute treatment of migraine with or without aura in adults, and a post hoc analysis presented at CMSC that demonstrated the effect of cladribine on the reduction of MRI lesions across subgroups in ORACLE-MS study (Transcript below).
Jenna: Welcome to Neurology News Network. I’m Jenna Payesko.Let’s get into the news from this week.
The FDA has approved Zolgensma for the treatment of spinal muscular atrophy in pediatric patients aged 2 and younger with mutations in the SMN1 gene. This is the first and only gene therapy FDA-approved for SMA, including patients who are presymptomatic.
Novartis, the parent company of AveXis, has announced access programs for Zolgensma, of which a single dose will be priced at $2.1 million dollars. AveXis is reportedly working closely with payers to offer pay-over-time options and affordability and access programs aimed at patients.
The FDA has granted De Novo clearance to Theranica’s Nerivio Migra, a smartphone-controlled electroceutical device that uses non-invasive neuromodulation for the acute treatment of migraine with or without aura in adults who do not have chronic migraine.
The device, a first-in-category product, is placed on the upper arm and utilizes smartphone-controlled electronic pulses to create a conditioned pain modulation response. The company is preparing to launch the device in the US market later this year.
Dr. Brian Grosberg, director of the Hartford Headache Center in Connecticut, who was the principal investigator on the TCH-003 trial, told NeurologyLive in an interview that “A device like this offers an opportunity for patients to not have to think about [medication use], and they don’t have to feel like they’re necessarily sacrificing efficacy.”
According to results of a post hoc analysis presented at the 2019 Annual Meeting of the Consortium of Multiple Sclerosis Centers in Seattle, Washington, the effect of cladribine on the reduction of the mean number of T1 gadolinium-enhancing lesions, active T2 lesions, and combined unique active lesions is consistent across subgroups of patients with a first demyelinating event who are at high risk of converting to multiple sclerosis. The subgroups were defined by baseline characteristics that are known to be potential modifiers of risk to conversion to clinically-definite multiple sclerosis, including age, gender, first classification of demyelinating event, presence of T1 gadolinium-enhancing lesions, and number of active T2 lesions.
The reduction in risk in lesion counts ranged from 60% to 89% for T1 gadolinium-enhancing lesions, 49% to 80% for active T2 lesions, and 61% to 83% for combined unique active lesions.
For more coverage of CMSC 2019 and other neurology news, head to neurologylive.com. This has been Neurology News Network. Thanks for watching.