Aducanumab BLA Accepted, Risdiplam Receives FDA Approval, Ofatumumab Lower ARRs Than Teriflunomide

This week Neurology News Network covered the FDA accepted biologics license application for aducanumab, FDA approval of risdiplam in patients with spinal muscular atrophy, and full data publication from the phase 3 ASCLEPIOS trials that compared ofatumumab and teriflunomide in patients with multiple sclerosis.

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.

Nearly 1 month after filing its biologics license application for its investigational Alzheimer disease treatment aducanumab, Biogen has announced that the FDA has accepted the application with priority review, setting a Prescription Drug User Fee Act target action date of March 7, 2021. Although the review is already on an accelerated track, the agency said it would act even earlier if the circumstances warrant it. The FDA also stated that it intends to soon hold an advisory committee meeting related to the application for the investigational human monoclonal antibody. If approved, aducanumab, previously known as BIIB037, would become the first treatment for reducing clinical decline in Alzheimer disease through its mechanism of selectively targeting aggregated forms of amyloid-beta, including both soluble oli-gomers and insoluble fibrils.

PTC Therapeutics announced that its investigational agent risidiplam has received FDA approval for the treatment of spinal muscular atrophy under the brand name Evrysdi. The agent is indicated for at-home oral administration of a liquid formulation in patients over the aged 2 months and older. The agency’s decision was made based on the findings of 2 clinical trials in risdiplam’s development, the FIREFISH and SUNFISH studies, both of which demonstrated the efficacy of the survival motor neuron 2-directed RNA splicing modifier in this patient population. Risdiplam-treated patients achieved key motor milestones, such as the ability to sit without support, and experienced preserved vital function and improved survival without the need for permanent ventilation.

According to the full data publication from the phase 3 ASCLEPIOS trials, among patients with multiple sclerosis, treatment with 20-mg subcutaneous injections of ofatumumab is associated with lower annualized relapse rates than with 14-mg oral tablet of teriflunomide (Aubagio; Sanofi). All told, the ARRs for patients with MS were reduced by 51% in the ACLEPIOS I and II trials, respectively, with both differences achieving statistical significance. “The ASCLEPIOS studies found that ofatumumab produced a significant reduction in new inflammation, as well as fewer clinical relapses and progression events,” said Stephen L. Hauser, MD, director, UCSF Weill Institute for Neurosciences; professor of neurology, UCSF School of Medicine; and co-chair of the steering committee for the ASCLEPIOS I and II studies, in a statement. “Ofatumumab represents a potential new option for RMS patients with greater efficacy compared to teriflunomide, a comparable safety profile, and the convenience of once monthly self-administration without the need for infusions.”

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