This week Neurology News Network covered the FDA approval of ozanimod for the treatment of adults with relapsing forms of MS, as well as the phase 1 results of intrathecal Zolgensma. Lastly, we touch on the recent new drug application for Jazz Pharmaceuticals' JZP-258. 

Marco Meglio: Welcome to this special edition of Neurology News Network. I’m Marco Meglio. Please excuse our appearance this week as a majority of the US workforce, including the NeurologyLive team, moves to working remote as we come together to help reduce the spread of the novel coronavirus.

The FDA has approved ozanimod for the treatment of adults with relapsing forms of multiple sclerosis, including clinically isolated syndrome, relapsing-remitting MS, and active secondary progressive MS. The oral S1P receptor modulator will be marketed as Zeposia. Although the drug requires no CYP2C9 genotyping or first-dose observation, it is suggested that an up-titration scheme be employed in order to reach a maintenance dose due to the risk of transient cardiac conduction issues. In addition to reduced Adjusted Relapse Rate, treatment with ozanimod was associated with a 63% relative reduction of T1-weighted gadolinium-enhanced lesions, and a 48% relative reduction in new or enlarging T2 lesions at 1 year in SUNBEAM.

Interim data from the phase 1 STRONG study of AveXis’s intrathecal Zolgensma in sitting but nonambulatory patients with spinal muscular atrophy demonstrated significant change in Hammersmith Functional Motor Scale-Expanded score from baseline to 12 months postdose. The FDA previously approved Zolgensma in May 2019 for the treatment of SMA in pediatric patients 2 years or less of age with mutations in the SMN1 gene. Zolgensma is the first and only gene therapy FDA-approved for SMA, including for patients who are presymptomatic. Researchers also noted that 92% of patients who received dose B achieved a clinically meaningful >3-point increase in HFMSE at any post-baseline visit during the study period. Additionally, 4 motor milestones were achieved in the same group, but the secondary end point of the ability to walk independently for >5 steps was not achieved by any patient.  

The FDA has accepted a new drug application for Jazz Pharmaceuticals’ JZP-258, an investigational, novel oxybate agent for the treatment of cataplexy and excessive daytime sleepiness in those 7 years and older with narcolepsy. Currently, the only approved agent for this indication is sodium oxybate, which has 92% more sodium, according to Jazz. During the double-blind withdrawal period, participants randomized to placebo experienced 2.35 weekly cataplexy attacks compared to 0 in the JZP-258 group. Additionally, significant increases in median ESS scores were observed in the placebo group compared to the treatment group. The agency has set the PDUFA action date for July 21, 2020.

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