Fenfluramine FDA Approved for Dravet Syndrome, Insulin Shows No Impact in Alzheimer Disease, MS Patients Develop More Severe COVID-19 Conditions

July 4, 2020

Neurology News Network for the week ending July 4, 2020.

This week Neurology News Network covered the FDA approval of fenfluramine as a treatment for patients with dravet sydrome, a study examining the benefits of insulin in patients with Alzheimer disease, and the risk factors involved in patients with multiple sclerosis during the COVID-19 pandemic.

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.

After pushing back its decision day by 3 months to review additional data, the FDA has approved fenfluramine for the treatment of Dravet syndrome. The rare, pediatric epilepsy syndrome characterized by frequent and severe refractory seizures is typically addressed with a cocktail of anticonvulsants, and more recently with GW Pharmaceutical’s cannabidiol therapy, Epidiolex. Fenfluramine will be available as an oral solution for patients age 2 and older. The medication does include a boxed warning for risk of valvular heart disease and pulmonary arterial hypertension, and will be distributed through the Fintepla Risk Evaluation and Mitigation Strategy Program by the end of July 2020. “There remains a huge unmet need for the many Dravet syndrome patients who continue to experience frequent severe seizures even while taking one or more of the currently available antiseizure medications,” said Joseph Sullivan, director of the Pediatric Epilepsy Center of Excellence at the UCSF Benioff Children’s Hospitals.

Data from a randomized, double-blind, phase 2/3 clinical trial revealed that treatment with intranasal insulin demonstrated no cognitive or functional benefits over a 12-month period in patients with mild cognitive impairment or Alzheimer disease dementia. The study consisted of 2 treatment groups that received 40 IU of intranasal insulin or placebo daily for 12 months using 2 different treatment devices, both administered intranasally, followed by a 6-month open-label extension phase. From baseline to month 12, the researchers concluded that no differences were observed between treatment arms in the mean score change on the Alzheimer Disease Assessment Scale—cognitive subscale 12 in the device 2 ITT cohort. Other outcome measures such as the Clinical Dementia Rating scale Sum of Boxes, Alzheimer Disease Cooperative Study Activities of Daily Living Scale for Mild Cognitive Impairment, and memory composite tests did not show any significant differences among treatment arms.

In a recent attempt to understand the risk factors involved for patients with multiple sclerosis (MS) to possibly develop a severe form of the novel COVID-19 infection, results suggest that neurological disability, age, and obesity were all associated with severe infection. No link was observed between exposure to disease-modifying therapies and COVID-19 severity. Infection severity was assessed on a 7-point ordinal scale ranging from 1—denoting no hospitalization with no limitations on activities—to 7—denoting death. There was a cutoff at 3 on the scale, which was defined as hospitalized and not requiring supplemental oxygen. All told, 21% of patients had a severity score of ≥3, and 3.5% died from COVID-19. A higher proportion of those with a severity score of ≥3 had no DMT exposure (46%) compared to those with DMT exposure.

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