Diroximel Fumarate Approved for MS; Personalized Interventions Improve Cognition; Zolgensma Trial Placed on Partial Hold

This week Neurology News Network covered the FDA approval of diroximel fumerate for the treatment of relapsing forms of MS, including relapsing-remitting and secondary progressive disease, as well as clinically isolated syndrome; new research showing that individually tailored interventions may help improve cognition and reduce Alzheimer dementia risk; and the partial FDA hold placed on a phase 1 clinical trial of intrathecal Zolgensma for the treatment of SMA type 2.

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

The FDA has approved multiple sclerosis agent diroximel fumarate for the treatment of relapsing forms of MS, including relapsing-remitting and secondary progressive disease, as well as clinically isolated syndrome. The agent’s new drug application was supported by findings from the EVOLVE-MS-1 study, an open-label phase 3 safety trial of the therapy in approximately 500 patients with relapsing-remitting MS. Biogen holds the exclusive, worldwide license to commercialize diroximel fumarate and issued its intention to make it available in the United States in the near future.

The therapeutic treatment of Alzheimer disease has proven to be extremely challenging. Amid numerous failed attempts to prevent disease progression and reduce the risk of disease development, the use of personalized, multidomain interventions have emerged as a promising new paradigm. Study results published in Alzheimer’s & Dementia suggest that adherence to individually tailored interventions, from improved diet and exercise to optimized blood pressure and blood sugar control, may improve cognition as well as reduce risk scores for Alzheimer’s disease in those at risk of developing Alzheimer’s disease dementia. The results place extra emphasis on the importance of early intervention and good adherence, with data demonstrating that higher compliance in patients across the spectrum, from cognitively normal to those with mild cognitive impairment due to Alzheimer’s Disease, resulted in significantly better improvement in cognition than that seen in historical cohorts. Lead author Richard Isaacson told NeurologyLive that the key finding of the study which he and his colleagues found “truly surprising” was the improvement in cognition over 18 months for patients in the early treatment cohort who were highly compliant. He said, “I think most people in the field would ask, ‘Well, is that really possible?’ And it was,”

Novartis’s tussle with the FDA appears to be far from over. After receiving a scathing letter from the agency back in August regarding data manipulation in preclinical assessments of Zolgensma, the FDA has now placed a partial hold on an ongoing clinical trial assessing intrathecal administration of the drug in patients with spinal muscular atrophy. The announcement comes after AveXis disclosed findings from a small, preclinical study that showed dorsal root ganglia mononuclear cell inflammation, which was sometimes accompanied by neuronal cell degeneration or loss. Notably, this partial hold by the agency does not affect the intravenous form of Zolgensma, an adeno-associated viral vector-based gene therapy, that is currently on the market or ongoing intravenous clinical trials.

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