Valbenazine Shines in Open-Label Extension, FDA Grants Breakthrough Designation to NfL Test, Tocilizumab Effective in NMOSD

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Neurology News Network for the week ending November 18, 2023. [WATCH TIME: 3 minutes]

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Welcome to this special edition of Neurology News Network. I’m Marco Meglio.

Months after the FDA approved valbenazine for the treatment of chorea associated with Huntington disease (HD), new interim data from the open-label extension (OLE) of the phase 3 KINECT-HD2 study continued to highlight the therapy’s longterm efficacy and safety. All told, improvements in chorea were observed at the first evaluation (week 2) when participants were taking the lowest dose of 40 mg, with efficacy sustained through week 50 at a maximal dose of 80 mg. Of 127 participants at the time of the analysis, 98 were from KINECT-HD. All told, mean TMC score reductions were observed by week 2 with valbenazine 40 mg and sustained with maximal doses of 80 mg from week 8 to week 50.

Recently, the FDA granted breakthrough device designation to Roche for its Elecsys Neurofilament Light Chain (NfL) test as a potential tool for the detection of disease activity in patients with multiple sclerosis (MS). NfL, a neuron-specific cytoskeletal protein that is released into the extracellular fluid following axonal injury, has been identified as a biomarker of disease activity in MS. Measurements of NfL levels can capture the extent of neuroaxonal damage, especially in the early stages of the disease. The Elecsys NfL test is intended for adults with MS aged 18 to 55 years old who have either relapsing-remitting or progressive forms of the disease. Although the current spotlight for NfL’s intended use is MS, increases in NfL concentrations have been reported in a wide range of neurologic conditions including Alzheimer disease, amyotrophic lateral sclerosis (ALS), Huntington disease, and more.

Data from a recently published meta-analysis of studies assessing tocilizumab found the therapy to be effective in reducing the frequency of relapses and improving patient function among those with neuromyelitis optica spectrum disorder (NMOSD). The effectiveness of the IL-6 inhibitor in reducing relapse rates was related to multiple factors such as gender, race, and dosage level. After searching MEDLINE, PubMed, Embase, and the Cochrane Library, investigators included 9 articles comprising 153 patients in the systematic review and meta-analysis. At the outset of treatment, the average age of all patient was 44.1 (range, 23-62) years, with most patients seropositive. Among those in the cohort, dosages of tocilizumab 8 mg/kg every 4 weeks were the most commonly used. After initiating therapy, findings showed a mean reduction of –0.81 in Expanded Disability Status Scale (EDSS) scores, with moderate heterogeneity detected.

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