Aducanumab BLA Submitted, Daridorexant Shows Phase 3 Efficacy, Neurologic Symptoms in Children With COVID-19

This week Neurology News Network covered the newly submitted biologics license application of aducanumab for the treatment of Alzheimer disease, the phase 3 study of daridorexant in elderly patients with insomnia, and one of the first case-series studies to evaluate children with COVID-19.

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.

Biogen announced that following a planned meeting with the FDA, it has completed and submitted a biologics license application for its investigational agent aducanumab for the treatment of Alzheimer disease. The BLA submission is supported by data from the phase 3 EMERGE and ENGAGE clinical trials, in addition to the phase 1b PRIME study. Biogen noted that it has requested a priority review designation, as if approved, aducanumab would become the first treatment for the reduction of clinical decline in Alzheimer, as well as the first to demonstrate clinical benefit through amyloid-beta removal. The BLA submission will be reviewed by the FDA and a decision returned in the next 60 days. Biogen noted that it is also continuing dialogue with other regulatory agencies in Europe and Japan.

The safety and efficacy of the dual orexin receptor antagonist daridorexant have been confirmed in a second pivotal phase 3 study in 924 adults and elderly patients with insomnia. The results follow a data readout in April 2020 from Idorsia’s first pivotal phase 3 study of daridorexant, which also showed promising results. The latest data demonstrate a statistically significant improvement in sleep maintenance and subjective total sleep time at months 1 and 3 for patients, nearly 40% of whom were 65 or older, who received the 25-mg dose. The therapy was also associated with positive effects on daytime functioning, as measured daily using the sleepiness domain score from the Insomnia Daytime Symptoms and Impacts Questionnaire, with participants reporting no residual morning sleepiness and no evidence of rebound or withdrawal symptoms after discontinuing treatment.

Results from a case-series study involving 27 children with COVID-19 pediatric multisystem inflammatory syndrome showed that a small subset of participants presented with new neurological symptoms involving both the central and peripheral nervous systems and splenial changes on imaging. Among the patient cohort observed in the study, 4 (14.8%) who were previously healthy had new-onset neurological symptoms. The symptoms documented were encephalopathy, headaches, brainstem and cerebellar signs, muscle weakness, and reduced reflexes. All 4 of the patients needed intensive care unit admission for the treatment of their COVID-19 pediatric multisystem inflammatory syndrome. Their ICU stays lasted a median 6.5 days, and mechanical ventilation duration was a median of 5 days. Additionally, MRI revealed splenium signal changes in all 4 of the patients.

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