Neurology News Network for the week ending May 7, 2022. [WATCH TIME: 4 minutes]
WATCH TIME: 4 minutes
Treatment with risdiplam (Evrysdi; Genentech), an FDA-approved agent for spinal muscular atrophy (SMA), was found to be safe and resulted in high rates of infants remaining alive at 3 years, according to newly announced data from the pivotal FIREFISH study (NCT02913482) and its open-label extension. Infants on the study drug showed continued improvement or maintained motor functions, including the ability to swallow, sit without support, stand with support, and walk while holding on, between 2 and 3 years of treatment.In total, 91% of infants (n = 58) on risdiplam were alive after 3 years of treatment. Among the 48 infants who had an available motor assessment, 32 infants maintained and 4 gained the ability to sit without support for at least 5 seconds since month 24, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development-III. Risdiplam, a survival motor neuron 2 splicing modifier, helped decrease the rate of hospitalizations from 1.24 per patient year over 12 months to 0.70 per patient year over 36 months. Notably, no additional deaths occurred since the primary analysis of FIREFISH, up to the data cut-off of this analysis.
Axsome Therapeutics has been issued a complete response letter (CRL) for its new drug application (NDA) for its investigational acute migraine treatment AXS-07. The company noted in an announcement that it neither identified nor raised any concerns regarding the efficacy or safety data in the application.The FDA additionally did not request any new clinical trials to be conducted. The principal reasons for the CRL were related to chemistry, manufacturing, and controls (CMC) considerations, specifically a requirement of further CMC data pertaining to the drug product and manufacturing process. Axsome noted that it will be able to address these concerns and plans to provide timing for a resubmission following a meeting with the agency. The NDA was supported by data from 2 phase 3 randomized controlled clinical trials—the MOMENTUM trial and the INTERCEPT trial—included in the new drug application, which was accepted for review in September 2021. The data demonstrate a statistically significant elimination of migraine pain with AXS-07 compared with placebo and active controls.
In a new post hoc analysis of the phase 3 EXPAND study (NCT01665144), investigators obtained and validated different responder profiles (RSP) of patients with multiple sclerosis (MS) to a disease-modifying therapy (DMT) using baseline characteristics that were associated with higher treatment effects. In conclusion, cognitive test deficits at baseline were predictive of further deterioration in the course of follow-up. Overall, 78% (1290 of 1645) of patients were pronounced responders to treatment with siponimod (Mayzent; Novartis), the study drug assessed, on at least 1 of the 4 clinical outcomes. "Cognitive assessment seems to be an important clinical sign of the severity of the disease [MS], not only on a cross-sectional level, but also if we look in the following years,” investigator Ludwig Kappos, MD, FEAN, FAAN, professor of neurology, University of Basel, told NeurologyLive®. EXPAND was a randomized, double-blind, placebo-controlled clinical trial that evaluated siponimod, an FDA-approved DMT, in a cohort of patients with progressive MS. The generated score was based off treatment effects on outcome measures such as Expanded Disability Status Scale (EDSS), Timed 25-Foot Walk (T25FW), 9-Hole Peg Test (9HPT), and Symbol Digit Modalities Test.
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