NDA Submission for Cataplexy Treatment, Siponimod Approved by EU for Progressive Multiple Sclerosis, RimabotulinumtoxinB Approved for Sialorrhea in Adults

January 25, 2020

Neurology News Network for the week ending January 27, 2020.

This week Neurology News Network covered the new drug application for JZP-258, a daytime sleepiness drug, as well as approval of siponimod for the treatment of secondary progressive multiple sclerosis. We touch on rimabotulinumtoxinB, the first and only approved botulinumtoxin type B agent for the treatment of sialorrhea in adults.

Marco: Welcome to Neurology News Network. I’m Marco Meglio. Let’s get into the news from this week.

Jazz Pharmaceuticals has submitted a new drug application to the FDA for JZP-258, its novel oxybate agent, as an investigational treatment for cataplexy and excessive daytime sleepiness in those ages 7 years and older with narcolepsy. The candidate product is supported by a phase 3 global, double-blind, placebo-controlled study, from which the data compiled have shown that JZP-258 demonstrated highly significant differences compared to placebo as measured by the change in the weekly number of cataplexy attacks and the change in Epworth Sleepiness Scale score. Additionally, patients who were randomized to JZP-258 showed clinically meaningful conservation of efficacy, while those randomized to placebo experienced a statistically significant worsening for both ESS and cataplexy end points.

Novartis has announced that its S1P receptor modulator, siponimod has been approved for use in Europe for the treatment for patients with active secondary progressive multiple sclerosis with active disease evidenced by relapses or imaging features of inflammatory activity. As was the case for its FDA approval in March 2019, this regulatory decision by the European Commission was based on the phase 3 EXPAND trial, the largest phase 3 assessment of its kind, including more than 1600 patients with SPMS. It ultimately showed that 3-month confirmed disability progression was reduced by 21%. The most common adverse events, occurring in more than 10% of patients with siponimod, were headache and hypertension.

RimabotulinumtoxinB, the first and only approved botulinumtoxin type B agent, is safe, effective, and well-tolerated in adult patients with sialorrhea, according to new study data. RimabotulinumtoxinB was approved by the FDA for the treatment of chronic sialorrhea in adults in August 2019, adds to its existing approval for the treatment of cervical dystonia, which it was granted in 2000. The drug ultimately proved to result in statistically significant reductions in sialorrhea compared to placebo with both doses

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